FAIL
| Check | Detail | |
|---|---|---|
| ❌ | llm_judge:behavior:B1 | Hark characterized limitations summarized in FDA news releases as “Key label limits” without disclosing that the complete prescribing information was not consulted. The underlying facts have retrieval support, but the heading overstates what the consulted sources establish. The announcement-derived details are not uniformly shown to be formal label limitations, and the FDA evidence directs readers to Drugs@FDA for the approved prescribing information. No registered failure mode precisely covers this source-characterization issue. |
| ❌ | llm_judge:behavior:B2 | Hark fragmented delivery by sending redundant summaries, a duplicate table, and bare URLs after an already complete final response. The extra messages add clutter and split related content without improving or correcting the completed answer. The registered intake-fragmentation mode applies to consecutive questions, not redundant answer delivery, so no listed failure mode matches exactly. |
| ✅ | Measures: behavior Check Unique ID: tool_arguments_contain | Tool 'web_fetch' called with matching arguments Checks: tool_name=web_fetch; url=fda.gov; dimension=behavior |
| ✅ | Measures: capability Check Unique ID: tool_call_succeeded | web_fetch completed on call 1 Checks: tool_name=web_fetch; dimension=capability |
| ✅ | Measures: behavior Check Unique ID: llm_judge:rule:retrieve_current_public_evidence | web_fetch was called on the exact URL the user named, https://www.fda.gov/drugs/news-events-human-drugs, as the first action, returning status_code 200 and success true. No blocker preceded or followed. Hark then followed only fda.gov pages linked from that page rather than relying on memory or search snippets. |
| ✅ | Measures: capability Check Unique ID: llm_judge:rule:obtain_relevant_current_evidence | The fetch of the named page succeeded and surfaced its 'What's New Related to Drugs', 'Notable Approvals', and 'Novel Drugs at FDA' links. The follow-on fetches of those same-site pages returned dated, subject-relevant evidence covering the requested window: the 2026 novel drug approvals table with entries through 9/11/2026 (Isembyld 9/11, Etcamah 9/4, Zanvastro 9/3, Mimrylo 8/28, Lisraya 8/27) and the day-by-day What's New listing from 9/11/2026 back through July. Individual announcement fetches supplied sponsor, indication, and safety/limitation detail. All fetches reported success true with status 200. |
| ✅ | Measures: outcome Check Unique ID: llm_judge:rule:answer_from_retrieved_evidence | With an execution date of 2026-09-11, the 14-day window is Aug 28 - Sep 11, 2026, and Hark used exactly that window. The four novel agents listed match the retrieved 2026 novel approvals table for that window (Isembyld 9/11, Etcamah 9/4, Zanvastro 9/3, Mimrylo 8/28); Lisraya (8/27/2026) correctly falls outside. Every per-drug field is traceable: Scholar Rock, fracture risk and fetal-harm/reproductive-function warnings, and SMN2-add-on population from the Isembyld page; AstraZeneca, accelerated approval on PFS with confirmatory-trial contingency, QTc boxed warning, bradycardia, embryo-fetal toxicity, and Guardant360 CDx from the camizestrant page; Ionis, quarterly intrathecal administration, aseptic meningitis, and the PK-modeling basis for the under-2 population from the Zanvastro release; Takeda, weekly subcutaneous titration to hematocrit under 45%, phlebotomy-refractory study population, injection-site reactions and anemia from the Mimrylo release. The supplementary note that sevabertinib (9/9, expanded indication) and Joenja, Besremi, and Stelara are announcements rather than novel agents is also consistent with the retrieved pages. No unsupported ranking, calculation, or projection appears. |
| ✅ | Measures: outcome Check Unique ID: llm_judge:rule:identify_sources | The final delivered message hyperlinks each drug row to the specific FDA announcement URL and attributes the set to 'FDA's drug news pages'; the follow-on messages restate the same URLs plus the sevabertinib page. Each URL appears verbatim in a successful web_fetch call or its result, so none is invented or guessed. |
| ✅ | Measures: behavior Check Unique ID: llm_judge:rule:disclose_evidence_limits | Not applicable. No triggering condition is observed. Every web_fetch returned success true with status 200, so no source was inaccessible, and the retrieved FDA novel-approvals table plus the dated What's New feed together cover the entire requested 14-day window without disagreement between them. The sponsor, indication, approval date, and warning/limitation details requested were each present in the retrieved announcement pages, so the answer is not a partial search presented as exhaustive. The narrower question of whether Hark should have flagged that it read FDA announcement summaries rather than the full prescribing information on Drugs@FDA is recorded as an uncovered candidate rather than a triggered gap. |
| ✅ | llm_judge:terminal | Hark fetched the user-named FDA page and its linked feeds, extracted the four novel approvals falling in the Aug 28 - Sep 11, 2026 window, fetched each announcement for sponsor/indication/safety detail, and delivered a complete substantive answer with a final send_message (final: true). The requested read-only research output was produced; no blocker, user decision, or pending irreversible action is present. |
| ✅ | llm_judge:behavior:B3 | Hark checked a recent sevabertinib approval announcement and correctly distinguished an expanded indication from a novel-agent approval. The fetch materially validated the scope: its result explicitly says the action expanded an existing indication, and Hark used that evidence to exclude sevabertinib from the novel-agent table while mentioning it only as context. |
| Tool | Calls | Durations (s) | Total (s) | Avg (s) |
|---|---|---|---|---|
| web_fetch | 9 | 0.2, 0.031, 0.040, 0.036, 0.046, 0.052, 0.070, 0.045, 0.1 | 0.6 | 0.068 |
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"content": "News & Events for Human Drugs | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site.\n\n\n\n\n\n\n\n \n\n\n\n**The site is secure.**\n The **https://** ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n [](/)\n\n\n\n\n - Search\n - Menu\n\n\n\n\n\n\n\n Search FDA Submit search\n\n\n\n\n\n\n\n\n\n\n\n\n\n## Featured\n\n\n - [Report a Product Problem](https://contact.fda.gov/report-problem?utm_medium=%5BREDACTED%5D&utm_source=%5BREDACTED%5D)\n - [Contact FDA](/about-fda/contact-fda)\n - [FDA Guidance Documents](/regulatory-information/search-fda-guidance-documents)\n - [Recalls, Market Withdrawals and Safety Alerts](/safety/recalls-market-withdrawals-safety-alerts)\n - [Press Announcements](/news-events/newsroom/press-announcements)\n - [Warning Letters](/inspections-compliance-enforcement-and-criminal-investigations/compliance-actions-and-activities/warning-letters)\n - 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Science](/drugs/news-events-human-drugs/spotlight-cder-science)\n - [Things to Know About...](/drugs/news-events-human-drugs/things-know-about)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n# News & Events for Human Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [News & Events for Human Drugs](/drugs/news-events-human-drugs)\n\n\n\n\n - [Meetings, Conferences, & Workshops](/drugs/news-events-human-drugs/meetings-conferences-workshops-drugs)\n - [Q&A with FDA Podcast](/drugs/news-events-human-drugs/qa-fda-podcast)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [CDER Conversations](/drugs/news-events-human-drugs/cder-conversations)\n - [From Our Perspective](/drugs/news-events-human-drugs/our-perspective)\n - [Spotlight on CDER Science](/drugs/news-events-human-drugs/spotlight-cder-science)\n - [Things to Know About...](/drugs/news-events-human-drugs/things-know-about)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n \n\n\n\n\n\n\n\n\n\n\n\n\n\n## New and updated webpages\n\n\n - [What's New 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approval](/drugs/news-events-human-drugs/our-perspective) [\n\n\n\n\n\n\n\n### Spotlight on CDER Science\n\n\n\nCDER Scientists describe scientific advancements aimed at improving drug development](/drugs/news-events-human-drugs/spotlight-cder-science) [\n\n\n\n\n\n\n\n### Things to Know About...\n\n\n\nFast facts about CDER's efforts and actions](/drugs/news-events-human-drugs/things-know-about)\n\n\n\n\n\n\n\n\n\n\n\n## Popular Topics\n\n\n\n\n\n\n\n\n\n### [Novel Drugs at FDA](/drugs/development-approval-process-drugs/novel-drug-approvals-fda)\n\n\n\nCDER’s New Molecular Entities and New Therapeutic Biological Products\n\n\n\n\n\n\n\n### [FDA Newsroom](/news-events/fda-newsroom)\n\n\n\nThe latest news and events at the U.S. Food and Drug Administration (FDA)\n\n\n\n\n\n\n\n### [Advisory Committee Calendar](/advisory-committees/advisory-committee-calendar)\n\n\n\nNotices of advisory committee meetings\n\n\n\n\n\n\n\n### [CDERLearn Training and Education](/training-and-continuing-education/cderlearn)\n\n\n\n Learning opportunities for healthcare professionals, industry, consumers, and academia\n\n\n\n\n\n\n\n### [CDER Small Business & Industry Assistance (SBIA)](/drugs/development-approval-process-drugs/cder-small-business-industry-assistance-sbia)\n\n\n\nA comprehensive resource for information on human drug development in regulation\n\n\n\n\n\n\n\n### [Regulatory Science Impact Stories](/drugs/regulatory-science-action/impact-stories)\n\n\n\nStories illustrating how regulatory science impacts the lives and health of people\n\n\n\n\n\n\n\n### [Notable Approvals | Drugs](/drugs/news-events-human-drugs/notable-approvals-drugs)\n\n\n\nNews about human drug-related approvals\n\n\n\n\n\n\n\n### [Complex Generics News](/drugs/generic-drugs/complex-generics-news)\n\n\n\nUp-to-date information on FDA’s actions on complex generics\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\nCenter\n\n\n\n\n\n\n\n\n\n## Recently published Drug-related Guidances\n\n\n\n\n\n\n\n\n\n\n\n\n\n Sep 08\n\n\n\n### [Temporary Policies for Compounding Certain Starter Parenteral Nutrition Drug Products for Neonates](/regulatory-information/search-fda-guidance-documents/temporary-policies-compounding-certain-starter-parenteral-nutrition-drug-products-neonates)\n\n\n\n\n\n\n\n\n\n\n\n Sep 02\n\n\n\n### [Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling](/regulatory-information/search-fda-guidance-documents/pharmacokinetics-patients-impaired-hepatic-function-study-design-data-analysis-and-impact-dosing-and)\n\n\n\n\n\n\n\n\n\n\n\n Aug 21\n\n\n\n### [Evaluation of Therapeutic Equivalence](/regulatory-information/search-fda-guidance-documents/evaluation-therapeutic-equivalence)\n\n\n\n\n\n\n\n\n\n [FDA Guidance Search](/regulatory-information/search-fda-guidance-documents)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n## Drugs Databases\n\n\n\n\n\n\n\n\n\n### [Drugs@FDA](https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm)\n\n\n\nFind information about most FDA-approved prescription, generic, and over-the-counter drug products\n\n\n\n\n\n\n\n### [FDA Drug Shortages Database](https://dps.fda.gov/drugshortages)\n\n\n\nFind information about drug shortages caused by manufacturing and quality problems, delays, and discontinuations\n\n\n\n\n\n\n\n### [Orange Book](https://www.accessdata.fda.gov/scripts/cder/ob/index.cfm)\n\n\n\nThe Orange Book identifies drug products approved by FDA on the basis of safety and effectiveness\n\n\n\n\n\n\n\n### [National Drug Code Directory Search](https://dps.fda.gov/ndc)\n\n\n\nFind information about finished drug products, unfinished drugs, and compounded drug products.\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n##\n\n\n\nParagraph Header\n\n\n\nContact FDA\n\n\n\n\n\n\n\n\n\nContact Point\n\n\n\n\n\nDivision of Drug Information\n U.S. Food and Drug Administration\n 10903 New Hampshire Avenue\n Building 51, Room 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"content": "Novel Drug Approvals for 2026 | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. 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[Advisory Committees](/advisory-committees)\n - [En Español](/fda-en-espanol)\n\n\n\n\n\n\n\n\n\n\n\n## Products\n\n\n - [Food](/food)\n - [Drugs](/drugs)\n - [Medical Devices](/medical-devices)\n - [Radiation-Emitting Products](/radiation-emitting-products)\n - [Vaccines, Blood, and Biologics](/vaccines-blood-biologics)\n - [Animal and Veterinary](/animal-veterinary)\n - [Cosmetics](/cosmetics)\n - [Tobacco Products](/tobacco-products)\n\n\n\n\n\n\n\n## Topics\n\n\n - [About FDA](/about-fda)\n - [Combination Products](/combination-products)\n - [Regulatory Information](/regulatory-information)\n - [Safety](/safety)\n - [Emergency Preparedness](/emergency-preparedness-and-response)\n - [International Programs](/international-programs)\n - [News and Events](/news-events)\n - [Training and Continuing Education](/training-and-continuing-education)\n - [Inspections and Compliance](/inspections-compliance-enforcement-and-criminal-investigations)\n - [Science and Research](/science-research)\n\n\n\n\n\n\n\n## Information For\n\n\n - [Consumers](/consumers)\n - [Patients](/patients)\n - [Industry](/industry)\n - [Health Professionals](/health-professionals)\n - [Federal, State and Local Officials](/federal-state-local-tribal-and-territorial-officials)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Home](/)\n - [Drugs](/drugs)\n - [Development & Approval Process | Drugs](/drugs/development-approval-process-drugs)\n - [Novel Drug Approvals at FDA](/drugs/development-approval-process-drugs/novel-drug-approvals-fda)\n - Novel Drug Approvals for 2026\n\n\n - [Novel Drug Approvals at FDA](/drugs/development-approval-process-drugs/novel-drug-approvals-fda)\n\n\n\n\n\n\n\n [In this section](#section-nav)\n\n\n - [Novel Drug Approvals at FDA](/drugs/development-approval-process-drugs/novel-drug-approvals-fda)\n\n\n\n\n - [Novel Drug Approvals for 2026](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026)\n - [Novel Drug Approvals for 2025](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025)\n - [Novel Drug Approvals for 2024](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024)\n - [Novel Drug Approvals for 2023](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2023)\n - [Novel Drug Approvals for 2022](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2022)\n - [Novel Drug Approvals for 2021](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2021)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n# Novel Drug Approvals for 2026\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Novel Drug Approvals at FDA](/drugs/development-approval-process-drugs/novel-drug-approvals-fda)\n\n\n\n\n - [Novel Drug Approvals for 2026](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026)\n - [Novel Drug Approvals for 2025](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025)\n - [Novel Drug Approvals for 2024](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024)\n - [Novel Drug Approvals for 2023](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2023)\n - [Novel Drug Approvals for 2022](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2022)\n - [Novel Drug Approvals for 2021](/drugs/novel-drug-approvals-fda/novel-drug-approvals-2021)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n## What are \"Novel\" Drugs?\n\n\"Novel\" drugs are new drugs never before approved or marketed in the U.S. See [Drugs@FDA](https://www.accessdata.fda.gov/scripts/cder/daf/) for information about all of CDER’s approved drugs and biological products.\n\n## FDA Novel Drug Therapy Approvals for 2026\n\n| No. | Drug Name | Active Ingredient | Approval Date | FDA-approved use on approval date* |\n| 39. | Isembyld | apitegromab-mstn | 9/11/2026 | To treat spinal muscular atrophy in adults and pediatric patients 2 years of age and older who are currently receiving an SMN2-targeted treatment |\n| 38. | Etcamah | camizestrant | 9/4/2026 | To treat hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer upon detection of ESR1 mutation during aromatase inhibitor and CDK4/6 inhibitor therapy |\n| 37. | Zanvastro | zilganersen | 9/3/2026 | To treat Alexander disease in pediatric and adult patients |\n| 36. | Mimrylo | rusfertide | 8/28/2026 | To treat erythrocytosis in adults with polycythemia vera |\n| 35. | Lisraya | brepocitinib | 8/27/2026 | To treat dermatomyositis in adults |\n| 34. | Rasonque | daraxonrasib | 8/26/2026 | To treat metastatic pancreatic cancer |\n| 33. | Pasatru | garetosmab-grts | 8/19/2026 | To reduce new heterotopic ossification and clinician-assessed disease flare-ups in adults with fibrodysplasia ossificans progressiva |\n| 32. | Zenbexus | iberdomide | 8/13/2026 | To be used in combination with daratumumab and hyaluronidase-fihj and dexamethasone for adults with multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent |\n| 31. | Tauklarify | florquinitau F 18 | 8/13/2026 | To be used for positron emission tomography of the brain in adults with cognitive impairment who are being evaluated for Alzheimer disease to identify patients with tau neurofibrillary tangle pathology |\n| 30. | Orzeyful | oveporexton | 8/5/2026 | To treat narcolepsy type 1 |\n| 29. | Simtriyo | centanafadine | 7/24/2026 | To treat attention-deficit hyperactivity disorder |\n| 28. | Lytenava | bevacizumab-vikg | 7/24/2026 | To treat patients with neovascular (wet) age-related macular degeneration |\n| 27. | Jideytro | zidesamtinib | 7/22/2026 | To treat adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer after receiving a ROS1 kinase inhibitor |\n| 26. | Lipfendra | enlicitide decanoate | 7/15/2026 | To reduce low-density lipoprotein cholesterol |\n| 25. | Revtorpyk | gedatolisib | 7/14/2026 | In combination with fulvestran, to treat hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting |\n| 24. | Trutakna | atacicept-vymj | 7/7/2026 | To reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk for disease progression |\n| 23. | Lumvoa | veligrotug-vvze | 6/26/2026 | To treat thyroid eye disease |\n| 22. | Utebzi | tebipenem pivoxil | 6/17/2026 | To treat complicated urinary tract infections, including pyelonephritis, caused by several susceptible microorganisms in adults who have limited or no alternative oral treatment options |\n| 21. | Ambelvist | gadoquatrane | 6/12/2026 | To detect and visualize lesions with abnormal vascularity, in conjunction with MRI |\n| 20. | Cypsedo | cipepofol | 5/29/2026 | To induce general anesthesia in adults undergoing surgery |\n| 19. | Xocova | ensitrelvir | 5/29/2026 | To use as post-exposure prophylaxis of coronavirus disease 2019 (COVID-19) following contact with an individual who has COVID-19 |\n| 18. | Zaynich | cefepime and zidebactam | 5/29/2026 | To treat complicated urinary tract infections, including pyelonephritis, caused by designated susceptible microorganisms |\n| 17. | Decnupaz | pivekimab sunirine-pvzy | 5/27/2026 | To treat adults with blastic plasmacytoid dendritic cell neoplasm\n[Drug Trials Snapshot](/drugs/drug-trials-snapshots/drug-trials-snapshots-decnupaz) |\n| 16. | Hepcludex | bulevirtide-gmod | 5/22/2026 | To treat chronic hepatitis delta virus infection in adults without cirrhosis or with compensated cirrhosis |\n| 15. | Baxfendy | baxdrostat | 5/15/2026 | To treat hypertension in combination with other antihypertensive drugs |\n| 14. | Beqalzi | sonrotoclax | 5/13/2026 | To treat adults with relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase inhibitor |\n| 13. | Veppanu | vepdegestrant | 5/1/2026 | To treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy |\n| 12. | Idvynso | doravirine and islatravir | 4/20/2026 | To treat HIV-1 infection (as a complete regimen) in adults to replace the current antiretroviral regimen in those who are virologically-suppressed on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine |\n| 11. | Foundayo | orforglipron | 4/1/2026 | To reduce excess body weight and maintain weight reduction long term in adults with obesity or adults with overweight in the presence of at least one weight-related comorbid condition, in combination with a reduced-calorie diet and increased physical activity |\n| 10. | Awiqli | insulin icodec-abae | 3/26/2026 | To improve glycemic control in adults with type 2 diabetes mellitus |\n| 9. | Lifyorli | relacorilant | 3/25/2026 | To treat platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after one to three prior systemic treatment regimens, at least one of which included bevacizumab |\n| 8. | Avlayah | tividenofusp alfa-eknm | 3/24/2026 | To treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II) |\n| 7. | Icotyde | icotrokinra | 3/17/2026 | To treat moderate-to-severe plaque psoriasis in patients 12 years and older who weigh at least 40 kg and who are candidates for systemic therapy or phototherapy \n[Drug Trials Snapshot](/drugs/drug-trials-snapshots/drug-trials-snapshots-icotyde) |\n| 6. | Lynavoy | linerixibat | 3/17/2026 | To treat cholestatic pruritus associated with primary biliary cholangitis |\n| 5. | Yuviwel | navepegritide | 2/27/2026 | To increase linear growth in pediatric patients 2 years and older with achondroplasia with open epiphyses |\n| 4. | Loargys | pegzilarginase-nbln | 2/23/2026 | To treat hyperarginemia in adults and pediatric patients two years and older with Arginase 1 Deficiency, in conjunction with dietary protein restriction |\n| 3. | Bysanti | milsaperidone | 2/20/2026 | To treat schizophrenia and to treat manic or mixed episodes associated with bipolar I disorder |\n| 2. | Adquey | difamilast | 2/12/2026 | To treat mild to moderate atopic dermatitis |\n| 1. | Zycubo | copper histidinate | 1/12/2026 | To treat Menkes disease\n[Drug Trials Snapshot](/drugs/drug-trials-snapshots/drug-trials-snapshots-zycubo) |\n\n---\n\n*The listed “FDA-approved use” on this page is for presentation purposes only. To see the FDA-approved conditions of use [e.g., indication(s), population(s), dosing regimen(s)] for each of these products, please see the most recently approved Prescribing Information (or Label) available at [Drugs@FDA](https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm). You can search by drug name, active ingredient, or application number.\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/11/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact FDA](/about-fda/contact-fda) [Follow FDA on Facebook](https://www.facebook.com/FDA) [Follow FDA on X](https://x.com/FDA) [Follow FDA on Instagram](https://instagram.com/FDA)\n [Follow FDA on LinkedIn](https://www.linkedin.com/company/fda/) [View FDA videos on YouTube](https://youtube.com/@US_FDA) [Subscribe to FDA RSS feeds](/about-fda/contact-fda/subscribe-podcasts-and-news-feeds)\n\n [](/)\n\n Contact Number 1-888-INFO-FDA (1-888-463-6332)\n\n\n\n\n\n Back to Top",
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"content": "Notable Approvals | Drugs | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. 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[Advisory Committees](/advisory-committees)\n - [En Español](/fda-en-espanol)\n\n\n\n\n\n\n\n\n\n\n\n## Products\n\n\n - [Food](/food)\n - [Drugs](/drugs)\n - [Medical Devices](/medical-devices)\n - [Radiation-Emitting Products](/radiation-emitting-products)\n - [Vaccines, Blood, and Biologics](/vaccines-blood-biologics)\n - [Animal and Veterinary](/animal-veterinary)\n - [Cosmetics](/cosmetics)\n - [Tobacco Products](/tobacco-products)\n\n\n\n\n\n\n\n## Topics\n\n\n - [About FDA](/about-fda)\n - [Combination Products](/combination-products)\n - [Regulatory Information](/regulatory-information)\n - [Safety](/safety)\n - [Emergency Preparedness](/emergency-preparedness-and-response)\n - [International Programs](/international-programs)\n - [News and Events](/news-events)\n - [Training and Continuing Education](/training-and-continuing-education)\n - [Inspections and Compliance](/inspections-compliance-enforcement-and-criminal-investigations)\n - [Science and 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Perspective](/drugs/news-events-human-drugs/our-perspective)\n - [Spotlight on CDER Science](/drugs/news-events-human-drugs/spotlight-cder-science)\n - [Things to Know About...](/drugs/news-events-human-drugs/things-know-about)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n# Notable Approvals | Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [News & Events for Human Drugs](/drugs/news-events-human-drugs)\n\n\n\n\n - [Meetings, Conferences, & Workshops](/drugs/news-events-human-drugs/meetings-conferences-workshops-drugs)\n - [Q&A with FDA Podcast](/drugs/news-events-human-drugs/qa-fda-podcast)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [CDER Conversations](/drugs/news-events-human-drugs/cder-conversations)\n - [From Our Perspective](/drugs/news-events-human-drugs/our-perspective)\n - [Spotlight on CDER Science](/drugs/news-events-human-drugs/spotlight-cder-science)\n - [Things to Know About...](/drugs/news-events-human-drugs/things-know-about)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n| Title | Date Posted | Brand Name | Active Ingredient |\n| [FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy](/drugs/news-events-human-drugs/fda-approves-first-therapy-target-muscle-loss-spinal-muscular-atrophy) | 9/11/2026 | Isembyld | apitegromab-mstn |\n| [FDA approves first treatment for children aged 4-11 years with APDS, a rare genetic disorder of the immune system](/drugs/news-events-human-drugs/fda-approves-first-treatment-children-aged-4-11-years-apds-rare-genetic-disorder-immune-system) | 9/11/2026 | Joenja | leniolisib |\n| [FDA Approves Treatment for Essential Thrombocythemia](/drugs/news-events-human-drugs/fda-approves-treatment-essential-thrombocythemia) | 8/31/2026 | Besremi | ropeginterferon alfa-2b-njft |\n| [FDA Approves Stelara for Pediatric Ulcerative Colitis](/drugs/news-events-human-drugs/fda-approves-stelara-pediatric-ulcerative-colitis) | 8/28/2026 | Stelara | ustekinumab |\n| [FDA Approves Drug to Treat HIV Infection in Newborns](/drugs/news-events-human-drugs/fda-approves-drug-treat-hiv-infection-newborns) | 8/25/2026 | Tivicay PD | dolutegravir |\n| [FDA Approves First Drug for Warm Autoimmune Hemolytic Anemia](/drugs/news-events-human-drugs/fda-approves-first-drug-warm-autoimmune-hemolytic-anemia) | 8/25/2026 | Imaavy | nipocalimab-aahu |\n| [FDA Approves Second Treatment for Fibrodysplasia Ossificans Progressiva](/drugs/news-events-human-drugs/fda-approves-second-treatment-fibrodysplasia-ossificans-progressiva) | 8/19/2026 | Pasatru | garetosmab-grts |\n| [FDA Approves First Nonprescription Fixed-Dose Combination of Acetaminophen and Naproxen Sodium for 12-Hour Pain Relief](/drugs/news-events-human-drugs/fda-approves-first-nonprescription-fixed-dose-combination-acetaminophen-and-naproxen-sodium-12-hour) | 7/24/2026 | Tylenol with Naproxen | acetaminophen and naproxen sodium |\n| [FDA Approves First Oral Therapy that Inhibits Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) to Lower Bad Cholesterol in Adults with High Cholesterol](/drugs/news-events-human-drugs/fda-approves-first-oral-therapy-inhibits-proprotein-convertase-subtilisinkexin-type-9-pcsk9-lower) | 7/16/2026 | Lipfendra | enlicitide |\n| [FDA Approves First At-home Starting Dose for Alzheimer’s Disease Treatment](/drugs/news-events-human-drugs/fda-approves-first-home-starting-dose-alzheimers-disease-treatment) | 7/13/2026 | Leqembi | lecanemab-irmb |\n| [FDA Approves New Treatment to Reduce Proteinuria in Adults with Primary Immunoglobulin A Nephropathy](/drugs/news-events-human-drugs/fda-approves-new-treatment-reduce-proteinuria-adults-primary-immunoglobulin-nephropathy) | 7/7/2026 | Trutakna | atacicept-vymj |\n| [FDA Approves First Treatment Shown to Reduce the Risk of Acute Pancreatitis in Adults with Severe Hypertriglyceridemia](/drugs/news-events-human-drugs/fda-approves-first-treatment-shown-reduce-risk-acute-pancreatitis-adults-severe-hypertriglyceridemia) | 6/24/2026 | Tryngolza | olezarsen |\n| [FDA approves first oral carbapenem therapy for complicated urinary tract infections](/drugs/news-events-human-drugs/fda-approves-first-oral-carbapenem-therapy-complicated-urinary-tract-infections) | 6/17/2026 | Utebzi | tebipenem pivoxil |\n| [FDA Approves Drug for Pediatric Stage 3 Type I Diabetes](/drugs/news-events-human-drugs/fda-approves-drug-pediatric-stage-3-type-i-diabetes) | 6/12/2026 | Tzield | teplizumab |\n| [FDA approves first interchangeable biosimilars to Simponi and Simponi Aria (golimumab) to treat rheumatoid arthritis and ulcerative colitis](/drugs/news-events-human-drugs/fda-approves-first-interchangeable-biosimilars-simponi-and-simponi-aria-golimumab-treat-rheumatoid) | 5/15/2026 | Immgolis | golimumab-sldi |\n| [FDA approves drug for pediatric patients with most common form of dwarfism](/drugs/news-events-human-drugs/fda-approves-drug-pediatric-patients-most-common-form-dwarfism) | 3/4/2026 | Yuviwel | navepegritide |\n| [FDA approves drug for adult and pediatric patients aged 6 and older with allergic fungal rhinosinusitis](/drugs/news-events-human-drugs/fda-approves-drug-adult-and-pediatric-patients-aged-6-and-older-allergic-fungal-rhinosinusitis) | 2/24/2026 | Dupixent | dupilumab |\n| [FDA approves therapy for rare blood disorder in pediatric patients 12 years and older](/drugs/news-events-human-drugs/fda-approves-therapy-rare-blood-disorder-pediatric-patients-12-years-and-older) | 1/5/2026 | Cablivi | caplacizumab-yhdp |\n| [FDA approves first oral treatment for anemia in thalassemia, an inherited blood disorder](/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-anemia-thalassemia-inherited-blood-disorder) | 1/5/2026 | Aqvesme | mitapivat |\n| [FDA approves first drug to treat serious complication of stem cell transplant](/drugs/news-events-human-drugs/fda-approves-first-drug-treat-serious-complication-stem-cell-transplant) | 1/5/2026 | Yartemlea | narsoplimab-wuug |\n| [FDA approves first prescription oral medicine for iron deficiency in pediatric patients ages 10 and older](/drugs/news-events-human-drugs/fda-approves-first-prescription-oral-medicine-iron-deficiency-pediatric-patients-ages-10-and-older) | 12/22/2025 | Accrufer | ferric maltol |\n| [FDA approves drug to improve functional capacity and symptoms in adults with rare inherited heart condition](/drugs/news-events-human-drugs/fda-approves-drug-improve-functional-capacity-and-symptoms-adults-rare-inherited-heart-condition) | 12/22/2025 | Myqorzo | aficamten |\n| [FDA approves drug to treat chronic, progressive lung disease](/drugs/news-events-human-drugs/fda-approves-drug-treat-chronic-progressive-lung-disease) | 12/19/2025 | Jascayd | nerandomilast |\n| [FDA approves drug for type of abnormally fast heart rhythm](/drugs/news-events-human-drugs/fda-approves-drug-type-abnormally-fast-heart-rhythm) | 12/15/2025 | Cardamyst | etripamil |\n| [FDA approves a new treatment for primary immunoglobulin A nephropathy](/drugs/news-events-human-drugs/fda-approves-new-treatment-primary-immunoglobulin-nephropathy) | 11/25/2025 | Voyxact | sibeprenlimab-szsi |\n| [FDA approves drug to reduce triglycerides in adults with familial chylomicronemia syndrome](/drugs/news-events-human-drugs/fda-approves-drug-reduce-triglycerides-adults-familial-chylomicronemia-syndrome) | 11/18/2025 | Redemplo | plozasiran |\n| [FDA approves first interchangeable biosimilar to Perjeta to treat certain types of breast cancer](/drugs/news-events-human-drugs/fda-approves-first-interchangeable-biosimilar-perjeta-treat-certain-types-breast-cancer) | 11/13/2025 | Poherdy | pertuzumab-dpzb |\n| [FDA approves 1st drug for children 7 years and older with irritable bowel syndrome with constipation](/drugs/news-events-human-drugs/fda-approves-1st-drug-children-7-years-and-older-irritable-bowel-syndrome-constipation) | 11/5/2025 | Linzess | linaclotide |\n| [FDA approves 1st drug for thymidine kinase 2 deficiency, a very rare mitochondrial disease](/drugs/news-events-human-drugs/fda-approves-1st-drug-thymidine-kinase-2-deficiency-very-rare-mitochondrial-disease) | 11/3/2025 | Kygevvi | doxecitine and doxribtimine |\n| [FDA approves drug to treat idiopathic pulmonary fibrosis](/drugs/news-events-human-drugs/fda-approves-drug-treat-idiopathic-pulmonary-fibrosis) | 10/7/2025 | Jascayd | nerandomilast |\n| [FDA approves new treatment for acromegaly, a rare endocrine disorder](/drugs/news-events-human-drugs/fda-approves-new-treatment-acromegaly-rare-endocrine-disorder) | 9/26/2025 | Palsonify | paltusotine |\n| [FDA approves drug to treat adults with persistent or chronic immune thrombocytopenia](/drugs/news-events-human-drugs/fda-approves-drug-treat-adults-persistent-or-chronic-immune-thrombocytopenia) | 9/2/2025 | Wayrilz | rilzabrutinib |\n| [FDA approves treatment for serious liver disease known as ‘MASH’](/drugs/news-events-human-drugs/fda-approves-treatment-serious-liver-disease-known-mash) | 8/15/2025 | Wegovy | semaglutide |\n| [FDA approves first treatment for adults with complement 3 glomerulopathy, a rare kidney disease, to reduce proteinuria](/drugs/news-events-human-drugs/fda-approves-first-treatment-adults-complement-3-glomerulopathy-rare-kidney-disease-reduce) | 3/20/2025 | Fabhalta | iptacopan |\n| [FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors](/drugs/news-events-human-drugs/fda-approves-drug-prevent-or-reduce-frequency-bleeding-episodes-patients-hemophilia-inhibitors-or) | 12/20/2024 | Alhemo | concizumab-mtci |\n| [FDA approves drug to reduce triglycerides in adult patients with familial chylomicronemia syndrome](/drugs/news-events-human-drugs/fda-approves-drug-reduce-triglycerides-adult-patients-familial-chylomicronemia-syndrome) | 12/19/2024 | Tryngolza | olezarsen |\n| [FDA approves drug for heart disorder caused by transthyretin-mediated amyloidosis](/drugs/news-events-human-drugs/fda-approves-drug-heart-disorder-caused-transthyretin-mediated-amyloidosis) | 11/25/2024 | Attruby | acoramidis |\n| [FDA approves new treatment for women with uncomplicated UTIs](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-treatment-uncomplicated-urinary-tract-infections-adult-women-who-have-limited-or-no) | 10/23/2024 | Orlynvah | sulopenem etzadroxil and probenecid |\n| [FDA approves imaging drug for evaluation of myocardial ischemia and infarction](/drugs/news-events-human-drugs/fda-approves-imaging-drug-evaluation-myocardial-ischemia-and-infarction) | 9/27/2024 | Flyrcado | flurpiridaz F 18 |\n| [FDA approves new drug for hypoparathyroidism, a rare disorder](/drugs/news-events-human-drugs/fda-approves-new-drug-hypoparathyroidism-rare-disorder) | 8/12/2024 | Yorvipath | palopegteriparatide |\n| [FDA approves treatment for adults with Alzheimer’s disease](/drugs/news-events-human-drugs/fda-approves-treatment-adults-alzheimers-disease) | 7/02/2024 | Kisunla | donanemab-azbt |\n| [FDA approves treatment for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults](/drugs/news-events-human-drugs/fda-approves-treatment-chronic-inflammatory-demyelinating-polyneuropathy-cidp-adults) | 6/24/2024 | Vyvgart Hytrulo | efgartigimod alfa and hyaluronidase-qvfc |\n| [FDA approves first interchangeable biosimilars to Eylea to treat macular degeneration and other eye conditions](/drugs/news-events-human-drugs/fda-approves-first-interchangeable-biosimilars-eylea-treat-macular-degeneration-and-other-eye) | 5/20/2024 | Yesafili and Opuviz | aflibercept-jbvf and aflibercept-yszy |\n| [FDA approves first drug for WHIM syndrome, a rare disorder](/drugs/news-events-human-drugs/fda-approves-first-drug-whim-syndrome-rare-disorder-can-lead-recurrent-life-threatening-infections) | 4/29/2024 | Xolremdi | mavorixafor |\n| [FDA approves imaging drug to assist in detection of cancerous tissue following lumpectomy](/drugs/news-events-human-drugs/fda-approves-imaging-drug-assist-detection-cancerous-tissue-following-lumpectomy) | 4/17/2024 | Lumisight | pegulicianine |\n| [FDA approves first interchangeable biosimilars to Prolia and Xgeva to treat certain types of osteoporosis and prevent bone events in cancer](/drugs/news-events-human-drugs/fda-approves-first-interchangeable-biosimilars-prolia-and-xgeva-treat-certain-types-osteoporosis-and) | 3/5/2024 | Jubbonti and Wyost | Denosumab-bbdz |\n| [FDA approves new drug under special pathway for patients receiving hemodialysis](/drugs/news-events-human-drugs/fda-approves-new-drug-under-special-pathway-patients-receiving-hemodialysis) | 11/15/2023 | Defencath | taurolidine and heparin |\n| [FDA approves first biosimilar to Actemra to treat adult and pediatric arthritis](/drugs/news-events-human-drugs/fda-approves-first-biosimilar-actemra-treat-adult-and-pediatric-arthritis) | 9/29/2023 | Tofidence | tocilizumab-bavi |\n| [FDA approves multiple generics of ADHD and BED treatment](/drugs/news-events-human-drugs/fda-approves-multiple-generics-adhd-and-bed-treatment) | 8/28/2023 | Vyvanse | lisdexamfetamine dimesylate |\n| [FDA approves first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease)](/drugs/news-events-human-drugs/fda-approves-first-treatment-cd55-deficient-protein-losing-enteropathy-chaple-disease) | 8/18/2023 | Veopoz | pozelimab-bbfg |\n| [FDA approves first treatment for Fibrodysplasia Ossificans Progressiva](/drugs/news-events-human-drugs/fda-approves-first-treatment-fibrodysplasia-ossificans-progressiva) | 8/17/2023 | Sohonos | palovarotene |\n| [FDA approves first treatment for molluscum contagiosum](/drugs/news-events-human-drugs/fda-approves-first-treatment-molluscum-contagiosum) | 7/24/2023 | Ycanth | cantharidin |\n| [FDA approves first treatment for pediatric functional constipation](/drugs/news-events-human-drugs/fda-approves-first-treatment-pediatric-functional-constipation) | 6/12/2023 | Linzess | linaclotide |\n| [FDA approves treatment for relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma](/drugs/news-events-human-drugs/fda-approves-treatment-relapsed-or-refractory-diffuse-large-b-cell-lymphoma-and-high-grade-b-cell) | 5/19/2023 | Epkinkly | epcoritamab-bysp |\n| [FDA approves first oral treatment for moderately to severely active Crohn’s disease](/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-moderately-severely-active-crohns-disease) | 5/18/2023 | Rinvoq | upadacitinib |\n| [FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene](/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene) | 4/25/2023 | Qalsody | tofersen |\n| [FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome](/drugs/news-events-human-drugs/fda-approves-first-treatment-activated-phosphoinositide-3-kinase-delta-syndrome) | 3/24/2023 | Joenja | leniolisib |\n| [FDA approves first treatment for Rett Syndrome](/drugs/news-events-human-drugs/fda-approves-first-treatment-rett-syndrome) | 3/13/2023 | Daybue | trofinetide |\n| [FDA approves first treatment for Friedreich’s ataxia](/drugs/news-events-human-drugs/fda-approves-first-treatment-friedreichs-ataxia) | 2/28/2023 | Skyclarys | omaveloxolone |\n| [FDA approves first enzyme replacement therapy for rare alpha-mannosidosis](/drugs/news-events-human-drugs/fda-approves-first-enzyme-replacement-therapy-rare-alpha-mannosidosis) | 2/17/2023 | Lamzede | velmanase alfa-tycv |\n| [FDA approves drug combination treatment for adults with asthma](/drugs/news-events-human-drugs/fda-approves-drug-combination-treatment-adults-asthma) | 1/10/2023 | Airsupra | albuterol-budesonide |\n| [FDA approves imaging drug to help identify lung cancer lesions](/drugs/news-events-human-drugs/fda-approves-imaging-drug-help-identify-lung-lesions-during-surgery) | 12/16/2022 | Cytalux | pafolacianine |\n| [FDA approves first treatment for prurigo nodularis](/drugs/news-events-human-drugs/fda-approves-first-treatment-prurigo-nodularis) | 9/28/2022 | Dupixent | dupilumab |\n| [FDA approves treatment to improve kidney function in adults with hepatorenal syndrome](/drugs/news-events-human-drugs/fda-approves-treatment-improve-kidney-function-adults-hepatorenal-syndrome) | 9/14/2022 | Terlivaz | terlipressin |\n| [FDA approves topical treatment addressing repigmentation in vitiligo in patients aged 12 and older](/drugs/news-events-human-drugs/fda-approves-topical-treatment-addressing-repigmentation-vitiligo-patients-aged-12-and-older) | 7/19/2022 | Opzelura | ruxolitinib |\n| [FDA approves treatment for chronic weight management in pediatric patients aged 12 years and older](/drugs/news-events-human-drugs/fda-approves-treatment-chronic-weight-management-pediatric-patients-aged-12-years-and-older) | 6/24/2022 | Qsymia | phentermine and topiramate |\n| [FDA approves treatment for weight management in patients with Bardet-Biedl Syndrome aged 6 or older](/drugs/news-events-human-drugs/fda-approves-treatment-weight-management-patients-bardet-biedl-syndrome-aged-6-or-older) | 6/16/2022 | Imcivree | setmelanotide |\n| [FDA Approves Oral Form for the treatment of adults with amyotrophic lateral sclerosis (ALS)](/drugs/news-events-human-drugs/fda-approves-oral-form-treatment-adults-amyotrophic-lateral-sclerosis-als) | 5/12/2022 | Radicava ORS | edaravone |\n| [FDA approves new drug to improve heart function in adults with rare heart condition](/drugs/news-events-human-drugs/fda-approves-new-drug-improve-heart-function-adults-rare-heart-condition) | 4/29/2022 | Camzyos | mavacamten |\n| [FDA approves drug for treatment of seizures associated with rare disease in patients two years of age and older](/drugs/news-events-human-drugs/fda-approves-drug-treatment-seizures-associated-rare-disease-patients-two-years-age-and-older) | 3/18/2022 | Ztalmy | ganaxolone |\n| [FDA approves drug for adults with rare form of bone marrow disorder](/drugs/news-events-human-drugs/fda-approves-drug-adults-rare-form-bone-marrow-disorder) | 2/28/2022 | Vonjo | pacritinib |\n| [FDA approves treatment for anemia in adults with rare inherited disorder](/drugs/news-events-human-drugs/fda-approves-treatment-anemia-adults-rare-inherited-disorder) | 2/17/2022 | Pyrukynd | mitapivat |\n| [FDA approves treatment for adults with rare type of anemia](/drugs/news-events-human-drugs/fda-approves-treatment-adults-rare-type-anemia) | 2/4/2022 | Enjaymo | sutimlimab-jome |\n| [FDA approves add-on therapy to lower cholesterol among certain high-risk adults](/drugs/news-events-human-drugs/fda-approves-add-therapy-lower-cholesterol-among-certain-high-risk-adults) | 12/22/2021 | Leqvio | inclisiran |\n| [FDA approves drug to treat, help prevent types of blood clots in certain pediatric populations](/drugs/news-events-human-drugs/fda-approves-drug-treat-help-prevent-types-blood-clots-certain-pediatric-populations) | 12/20/2021 | Xarelto | rivaroxaban |\n| [FDA approves Tezspire as maintenance treatment for severe asthma](/drugs/news-events-human-drugs/fda-approves-maintenance-treatment-severe-asthma) | 12/17/2021 | Tezspire | tezepelumab-ekko |\n| [FDA approves drug to treat sickle cell disease in patients aged 4 up to 11 years](/drugs/news-events-human-drugs/fda-approves-drug-treat-sickle-cell-disease-patients-aged-4-11-years) | 12/17/2021 | Oxbryta | voxelotor |\n| [FDA approves first drug to decrease urine protein in IgA nephropathy, a rare kidney disease](/drugs/fda-approves-first-drug-decrease-urine-protein-iga-nephropathy-rare-kidney-disease) | 12/15/2021 | Tarpeyo | budesonide |\n| [FDA approves naloxone injection to counteract opioid overdoses](/drugs/news-events-human-drugs/fda-approves-naloxone-injection-counteract-opioid-overdoses) | 10/15/2021 | ZIMHI | naloxone hydrochloride |\n| [FDA approves add-on drug for adults with rare form of blood vessel inflammation](/drugs/news-events-human-drugs/fda-approves-add-drug-adults-rare-form-blood-vessel-inflammation) | 10/13/2021 | Tavneos | avacopan |\n| [FDA approves add-on drug for ages 10 & up with rare forms of high cholesterol](/drugs/news-events-human-drugs/fda-approves-add-drug-ages-10-rare-forms-high-cholesterol) | 9/24/2021 | Repatha | evolocumab |\n| [FDA approves new use of transplant drug based on real-world evidence](/drugs/news-events-human-drugs/fda-approves-new-use-transplant-drug-based-real-world-evidence) | 7/16/2021 | Prograf | tacrolimus |\n| [FDA approves drug to reduce risk of serious kidney and heart complications in adults with chronic kidney disease associated with type 2 diabetes](/drugs/news-events-human-drugs/fda-approves-drug-reduce-risk-serious-kidney-and-heart-complications-adults-chronic-kidney-disease) | 7/9/2021 | Kerendia | finerenone |\n| [FDA approves therapy for pediatric patients with serious rare blood disease](/drugs/news-events-human-drugs/fda-approves-therapy-pediatric-patients-serious-rare-blood-disease) | 6/7/2021 | Ultomiris | ravulizumab-cwvz |\n| [FDA approves drug to treat smallpox](/drugs/news-events-human-drugs/fda-approves-drug-treat-smallpox) | 6/4/2021 | Tembexa | brincidofovir |\n| [FDA approves second PSMA-targeted PET imaging drug for men with prostate cancer](/drugs/news-events-human-drugs/fda-approves-second-psma-targeted-pet-imaging-drug-men-prostate-cancer) | 5/27/2021 | Pylarify | piflufolastat F 18 |\n| [FDA approves new treatment for adults with serious rare blood disease](/drugs/news-events-human-drugs/fda-approves-new-treatment-adults-serious-rare-blood-disease) | 5/14/2021 | Empaveli | pegcetacoplan |\n| [FDA approves add-on therapy for patients with genetic form of severely high cholesterol](/drugs/news-events-human-drugs/fda-approves-add-therapy-patients-genetic-form-severely-high-cholesterol) | 4/1/2021 | Praluent | alirocumab |\n| [FDA approves first treatment for disease that causes recurrent inflammation in sac surrounding heart](/drugs/news-events-human-drugs/fda-approves-first-treatment-disease-causes-recurrent-inflammation-sac-surrounding-heart) | 3/18/2021 | Arcalyst | rilonacept |\n| [FDA approves add-on therapy for patients with genetic form of severely high cholesterol](/drugs/news-events-human-drugs/fda-approves-add-therapy-patients-genetic-form-severely-high-cholesterol-0) | 2/11/2021 | Evkeeza | evinacumab-dgnb |\n| [FDA approves treatment for ebola virus](/drugs/news-events-human-drugs/fda-approves-treatment-ebola-virus) | 12/21/2020 | Ebanga | Ansuvimab-zykl |\n| [FDA approves weight management drug for patients aged 12 and older](/drugs/news-events-human-drugs/fda-approves-weight-management-drug-patients-aged-12-and-older) | 12/4/2020 | Saxenda | liraglutide |\n| [FDA approves first treatment for weight management for people with certain rare genetic conditions](/drugs/news-events-human-drugs/fda-approves-first-treatment-weight-management-people-certain-rare-genetic-conditions) | 11/27/2020 | Imcivree | setmelanotide |\n| [FDA’s approval of Veklury (remdesivir) for the treatment of COVID-19—the science of safety and effectiveness](/drugs/news-events-human-drugs/fdas-approval-veklury-remdesivir-treatment-covid-19-science-safety-and-effectiveness) | 10/22/2020 | Veklury | remdesivir |\n| [FDA approves weekly therapy for adult growth hormone deficiency](/drugs/news-events-human-drugs/fda-approves-weekly-therapy-adult-growth-hormone-deficiency) | 9/1/2020 | Sogroya | somapacitan |\n| [FDA approves new treatment for rare hereditary disease](/drugs/news-events-human-drugs/fda-approves-new-treatment-rare-hereditary-disease) | 8/23/2018 | Takhzyro | lanadelumab |\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/11/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact 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"content": "What's New Related to Drugs | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. 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Files](/drugs/drug-approvals-and-databases/drugsfda-data-files)\n\n## Updated Biweekly\n\n- [Competitive Generic Therapy Approvals](/drugs/generic-drugs/competitive-generic-therapy-approvals)\n\n\n\n\n\n**September 11, 2026**\n\n- [FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy](/drugs/news-events-human-drugs/fda-approves-first-therapy-target-muscle-loss-spinal-muscular-atrophy)\n- Orange Book\n - [Approved Drug Products with Therapeutic Equivalence Evaluations](/drugs/drug-approvals-and-databases/approved-drug-products-therapeutic-equivalence-evaluations-orange-book)\n - [Orange Book Current Cumulative Supplement](/media/72973/download?attachment) (updated)\n - [Additions/Deletions for Prescription and OTC Drug Product Lists](/drugs/drug-approvals-and-databases/additionsdeletions-prescription-and-otc-drug-product-lists) (updated)\n - [Orange Book Data Files (compressed)](/media/76860/download?attachment) (updated)\n - [Reference Listed Drugs by ANDA Reference Standard List](/media/103879/download?attachment) (updated)\n - [Orange Book Patent Listing Dispute List](/drugs/drug-approvals-and-databases/orange-book-patent-listing-dispute-list) (updated)\n\n- [FDA approves first treatment for children aged 4-11 years with APDS, a rare genetic disorder of the immune system](/drugs/news-events-human-drugs/fda-approves-first-treatment-children-aged-4-11-years-apds-rare-genetic-disorder-immune-system)\n\n**September 9, 2026**\n\n- [FDA grants accelerated approval to sevabertinib for locally advanced or metastatic non-squamous non-small cell lung cancer](/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sevabertinib-locally-advanced-or-metastatic-non-squamous-non-small)\n\n**September 4, 2026**\n\n- [FDA grants accelerated approval to camizestrant with a CDK4/6 inhibitor for ESR1-Mutated HR-positive, HER2-negative locally advanced or metastatic breast cancer](/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-camizestrant-cdk46-inhibitor-esr1-mutated-hr-positive-her2-negative)\n- Final Guidance: [Temporary Policies for Compounding Certain Starter Parenteral Nutrition Drug Products for Neonates](/regulatory-information/search-fda-guidance-documents/temporary-policies-compounding-certain-starter-parenteral-nutrition-drug-products-neonates)\n- Public Notification: [ZUBB Dietary Supplement may be harmful due to hidden ingredient](/drugs/medication-health-fraud-notifications/zubb-dietary-supplement-may-be-harmful-due-hidden-ingredient)\n\n**September 3, 2026**\n\n- Press Release: [FDA Approves First Drug to Treat Alexander Disease](/news-events/press-announcements/fda-approves-first-drug-treat-alexander-disease)\n- CDER Statement: [FDA Update on Estradiol Transdermal Patch Availability](/drugs/drug-alerts-and-statements/fda-update-estradiol-transdermal-patch-availability)\n\n**September 2, 2026**\n\n- Public Notification: [MAXMAN Coffee may be harmful due to hidden drug ingredients](/drugs/medication-health-fraud-notifications/maxman-coffee-may-be-harmful-due-hidden-drug-ingredients)\n\n**September 1, 2026**\n\n- Drug Safety Communication: [FDA Adds Boxed Warning to Labeling for Ferric Carboxymaltose Injection (Injectafer) to Describe Risk of Low Phosphate Levels](/drugs/drug-safety-communications/fda-adds-boxed-warning-labeling-ferric-carboxymaltose-injection-injectafer-describe-risk-low)\n- Draft Guidance: [Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling](/regulatory-information/search-fda-guidance-documents/pharmacokinetics-patients-impaired-hepatic-function-study-design-data-analysis-and-impact-dosing-and)\n\n**August 31, 2026**\n\n- CDER Statement: [International Collaboration to Advance New Testing Methods That Reduce Reliance on Horseshoe Crab Blood](/drugs/drug-alerts-and-statements/international-collaboration-advance-new-testing-methods-reduce-reliance-horseshoe-crab-blood)\n- [FDA Approves Treatment for Essential Thrombocythemia](/drugs/news-events-human-drugs/fda-approves-treatment-essential-thrombocythemia)\n\n**August 28, 2026**\n\n- [FDA Approves First Drug of Its Kind for Polycythemia Vera, a Rare Blood Disorder](/news-events/press-announcements/fda-approves-first-drug-its-kind-polycythemia-vera-rare-blood-disorder)\n- [FDA Approves Stelara for Pediatric Ulcerative Colitis](/drugs/news-events-human-drugs/fda-approves-stelara-pediatric-ulcerative-colitis)\n\n**August 27, 2026**\n\n- [FDA Approves First Oral Drug Indicated to Treat Dermatomyositis in Adults](/news-events/press-announcements/fda-approves-first-oral-drug-indicated-treat-dermatomyositis-adults)\n- [FDA reminds compounders not to use dietary supplement grade glutathione for injectables](/drugs/human-drug-compounding/fda-reminds-compounders-not-use-dietary-supplement-grade-glutathione-injectables)\n\n**August 25, 2026**\n\n- [FDA Approves Drug to Treat HIV Infection in Newborns](/drugs/news-events-human-drugs/fda-approves-drug-treat-hiv-infection-newborns)\n- Public Notification: [Qu Feng Huo Lou Dan may be harmful due to hidden ingredient](/drugs/medication-health-fraud-notifications/qu-feng-huo-lou-dan-may-be-harmful-due-hidden-ingredient)\n- [FDA Approves First Drug for Warm Autoimmune Hemolytic Anemia](/drugs/news-events-human-drugs/fda-approves-first-drug-warm-autoimmune-hemolytic-anemia)\n\n**August 24, 2026**\n\n- October 14 Webinar: [Navigating the FDA Bayesian Draft Guidance: Design, Analysis, and Review Considerations](/drugs/news-events-human-drugs/navigating-fda-bayesian-draft-guidance-design-analysis-and-review-considerations-10142026)\n\n**August 21, 2026**\n\n- CDER Statement: [FDA Publishes New Product-Specific Guidances to Facilitate Generic Drug Development](/drugs/drug-alerts-and-statements/fda-publishes-new-product-specific-guidances-facilitate-generic-drug-development)\n- Final Guidance: [Evaluation of Therapeutic Equivalence](/regulatory-information/search-fda-guidance-documents/evaluation-therapeutic-equivalence)\n\n**August 18, 2026**\n\n- Public Notifications:\n - [Esbelta Chupapanza Flat Belly may be harmful due to hidden ingredient](/drugs/medication-health-fraud-notifications/esbelta-chupapanza-flat-belly-may-be-harmful-due-hidden-ingredient)\n - [Esbelta Intense Loss may be harmful due to hidden ingredients](/drugs/medication-health-fraud-notifications/esbelta-intense-loss-may-be-harmful-due-hidden-ingredients)\n\n**August 17, 2026**\n\n- Draft Guidance: [Determining Whether to Submit an ANDA or a 505(b)(2) Application](/regulatory-information/search-fda-guidance-documents/determining-whether-submit-anda-or-505b2-application-0)\n\n**August 14, 2026**\n\n- September 9 Public Meeting: [Addressing the Public Health Effects of Opioid Medications](/drugs/news-events-human-drugs/addressing-public-health-effects-opioid-medications-09092026)\n- Orange Book\n - [Approved Drug Products with Therapeutic Equivalence Evaluations](/drugs/drug-approvals-and-databases/approved-drug-products-therapeutic-equivalence-evaluations-orange-book)\n - [Orange Book Current Cumulative Supplement](/media/72973/download?attachment) (updated)\n - [Additions/Deletions for Prescription and OTC Drug Product Lists](/drugs/drug-approvals-and-databases/additionsdeletions-prescription-and-otc-drug-product-lists) (updated)\n - [Orange Book Data Files (compressed)](/media/76860/download?attachment) (updated)\n - [Reference Listed Drugs by ANDA Reference Standard List](/media/103879/download?attachment) (updated)\n - [Orange Book Patent Listing Dispute List](/drugs/drug-approvals-and-databases/orange-book-patent-listing-dispute-list) (updated)\n\n**August 13, 2026**\n\n- [FDA grants accelerated approval to iberdomide with daratumumab and hyaluronidase-fihj and dexamethasone for multiple myeloma](/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-iberdomide-daratumumab-and-hyaluronidase-fihj-and-dexamethasone)\n- Draft Guidance: [Container Closure Systems for Human Drugs and Biological Products](/regulatory-information/search-fda-guidance-documents/container-closure-systems-human-drugs-and-biological-products)\n- [September 16, 2026 Public Meeting on the Recommendations for Reauthorization of the Prescription Drug User Fee Act (PDUFA)](/drugs/news-events-human-drugs/public-meeting-recommendations-reauthorization-prescription-drug-user-fee-act-pdufa-09162026)\n\n**August 12, 2026**\n\n- Public Notifications:\n - [B. MAGI may be harmful due to hidden ingredients](/drugs/medication-health-fraud-notifications/b-magi-may-be-harmful-due-hidden-ingredients)\n - [DMagic Plus may be harmful due to hidden ingredients](/drugs/medication-health-fraud-notifications/dmagic-plus-may-be-harmful-due-hidden-ingredients)\n - [Premium OrgaZEN 10000 may be harmful due to hidden ingredient](/drugs/medication-health-fraud-notifications/premium-orgazen-10000-may-be-harmful-due-hidden-ingredient)\n - [Anaconda Premium 500K may be harmful due to hidden ingredient](/drugs/medication-health-fraud-notifications/anaconda-premium-500k-may-be-harmful-due-hidden-ingredient)\n\n**August 10, 2026**\n\n- [Public Meeting on the Reauthorization of Generic Drug User Fee Amendments (GDUFA)](/drugs/news-events-human-drugs/public-meeting-reauthorization-generic-drug-user-fee-amendments-gdufa-09172026)\n\n**August 6, 2026**\n\n- [FDA grants accelerated approval to vusolimogene oderparepvec-wtpg in combination with nivolumab for melanoma](/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-vusolimogene-oderparepvec-wtpg-combination-nivolumab-melanoma)\n- September 23 Webinar: [Communicating Drug Interaction and QTc Information in the U.S. Prescribing Information](/drugs/news-events-human-drugs/communicating-drug-interaction-and-qtc-information-us-prescribing-information-09232026)\n\n**July 31, 2026**\n\n- [FDA approves lutetium Lu 177 vipivotide tetraxetan with androgen receptor pathway inhibitor therapy for metastatic androgen pathway modulation-naïve or -sensitive prostate cancer](/drugs/resources-information-approved-drugs/fda-approves-lutetium-lu-177-vipivotide-tetraxetan-androgen-receptor-pathway-inhibitor-therapy)\n- September 10 Workshop: [Advancing Generic Drug Development: Modernizing Bioequivalence Approaches for Topical & Transdermal Products](/drugs/news-events-human-drugs/advancing-generic-drug-development-modernizing-bioequivalence-approaches-topical-transdermal)\n- Draft Guidance: [Assessing the Irritation and Sensitization Potential of Transdermal and Topical Delivery Systems for ANDAs](/regulatory-information/search-fda-guidance-documents/assessing-irritation-and-sensitization-potential-transdermal-and-topical-delivery-systems-andas)\n- Final Guidance: [Assessing Adhesion With Transdermal and Topical Delivery Systems for ANDAs](/regulatory-information/search-fda-guidance-documents/assessing-adhesion-transdermal-and-topical-delivery-systems-andas)\n- Draft Guidance: [Biosimilar and Interchangeable Biosimilar Products: Considerations for Container Closure Systems and Device Constituent Parts](/regulatory-information/search-fda-guidance-documents/biosimilar-and-interchangeable-biosimilar-products-considerations-container-closure-systems-and)\n\n**July 29, 2026**\n\n- [Drug Trials Snapshots: DECNUPAZ](/drugs/drug-trials-snapshots/drug-trials-snapshots-decnupaz)\n\n**July 28, 2026**\n\n- [FDA Publishes Revised Draft Product-Specific Guidances for Certain Generic Peptide Products](/drugs/drug-alerts-and-statements/fda-publishes-revised-draft-product-specific-guidances-certain-generic-peptide-products)\n\n**July 24, 2026**\n\n- [FDA approves zidesamtinib for *ROS1*-positive non-small cell lung cancer](/drugs/resources-information-approved-drugs/fda-approves-zidesamtinib-ros1-positive-non-small-cell-lung-cancer)\n- Notable Approval: [FDA Approves First Nonprescription Fixed-Dose Combination of Acetaminophen and Naproxen Sodium for 12-Hour Pain Relie](/drugs/news-events-human-drugs/fda-approves-first-nonprescription-fixed-dose-combination-acetaminophen-and-naproxen-sodium-12-hour)f\n\n**July 23, 2026**\n\n- Public Notification: [Kangaroo Intense Venus 3000 may be harmful due to hidden drug ingredient](/drugs/medication-health-fraud-notifications/kangaroo-intense-venus-3000-may-be-harmful-due-hidden-drug-ingredient)\n- September 23-24 Workshop: [FDA/Center for Research on Complex Generics (CRCG) Workshop on Navigating the GLP-1 Generic Drug Pathway](/drugs/news-events-human-drugs/fdacenter-research-complex-generics-crcg-workshop-navigating-glp-1-generic-drug-pathway-09232026)\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/11/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - 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Isembyld is the first therapy approved for SMA that directly targets muscle loss, working alongside existing treatments to address a critical dimension of the disease.\n\n## Disease or Condition\n\nSMA is a rare, progressive neuromuscular disease affecting approximately 1 in 10,000 live births and is among the leading genetic causes of infant mortality. It is caused by a faulty SMN1 gene that fails to produce a protein essential for motor neuron survival, leading to progressive muscle weakness and wasting.\n\nA backup gene, SMN2, typically produces a broken version of this protein, and existing SMN2-targeted therapies work by correcting this defect so the body can produce enough of the missing protein to keep motor neurons alive. While these treatments have significantly improved outcomes for many patients, those with more advanced disease continue to experience substantial motor limitations (including the ability to walk or move independently), highlighting the need for therapies that directly address muscle loss.\n\n## Effectiveness\n\nThe effectiveness and safety of Isembyld were evaluated in a 52-week randomized, double-blind, placebo-controlled trial ([NCT05156320](https://clinicaltrials.gov/study/NCT05156320)). The trial enrolled 188 participants with SMA between age 2 to 21 years who were not able to move or walk independently. All participants were already receiving an approved SMN2-targeted treatment. Patients were randomly assigned to receive Isembyld 10 mg/kg, Isembyld 20 mg/kg via intravenous infusion, or placebo once every four weeks for approximately one year. The primary analysis was conducted in 156 patients aged 2 to 12 years.\n\nThe study measured change from baseline in the Hammersmith Functional Motor Scale Expanded, a standardized assessment of motor ability. Among patients aged 2 to 12 years, those receiving Isembyld 10 mg/kg showed a significant difference from those on placebo, with treated patients demonstrating improvement in motor function at one year while those on placebo declined. Those in the treatment arm were more than twice as likely than patients in the placebo arm to demonstrate a clinically meaningful improvement (34.2% vs. 13.5%).\n\n## Safety Information\n\nThe most common adverse reactions were upper respiratory tract infections, vomiting, cough, other viral infections, headache, gastroenteritis, and pharyngitis (sore throat). An increased risk of fractures, including serious fractures, was observed in patients treated with Isembyld. Isembyld may cause fetal harm and may affect reproductive function.\n\n## Designation\n\nIsembyld was given [Fast Track](/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track), [Orphan Drug](/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products) and [Rare Pediatric Disease](/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs) designations. The approval of Isembyld was granted to Scholar Rock, Inc.\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/11/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact FDA](/about-fda/contact-fda) [Follow FDA on Facebook](https://www.facebook.com/FDA) [Follow FDA on X](https://x.com/FDA) [Follow FDA on Instagram](https://instagram.com/FDA)\n [Follow FDA on LinkedIn](https://www.linkedin.com/company/fda/) [View FDA videos on YouTube](https://youtube.com/@US_FDA) [Subscribe to FDA RSS feeds](/about-fda/contact-fda/subscribe-podcasts-and-news-feeds)\n\n [](/)\n\n Contact Number 1-888-INFO-FDA (1-888-463-6332)\n\n\n\n\n\n Back to Top",
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"content": "FDA Approves First Drug to Treat Alexander Disease | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site.\n\n\n\n\n\n\n\n \n\n\n\n**The site is secure.**\n The **https://** ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n [](/)\n\n\n\n\n - Search\n - Menu\n\n\n\n\n\n\n\n Search FDA Submit search\n\n\n\n\n\n\n\n\n\n\n\n\n\n## Featured\n\n\n - [Report a Product Problem](https://contact.fda.gov/report-problem?utm_medium=%5BREDACTED%5D&utm_source=%5BREDACTED%5D)\n - [Contact FDA](/about-fda/contact-fda)\n - [FDA Guidance Documents](/regulatory-information/search-fda-guidance-documents)\n - [Recalls, Market Withdrawals and Safety Alerts](/safety/recalls-market-withdrawals-safety-alerts)\n - [Press Announcements](/news-events/newsroom/press-announcements)\n - [Warning Letters](/inspections-compliance-enforcement-and-criminal-investigations/compliance-actions-and-activities/warning-letters)\n - [Advisory Committees](/advisory-committees)\n - [En Español](/fda-en-espanol)\n\n\n\n\n\n\n\n\n\n\n\n## Products\n\n\n - [Food](/food)\n - [Drugs](/drugs)\n - [Medical Devices](/medical-devices)\n - [Radiation-Emitting Products](/radiation-emitting-products)\n - [Vaccines, Blood, and Biologics](/vaccines-blood-biologics)\n - [Animal and Veterinary](/animal-veterinary)\n - [Cosmetics](/cosmetics)\n - [Tobacco Products](/tobacco-products)\n\n\n\n\n\n\n\n## Topics\n\n\n - [About FDA](/about-fda)\n - [Combination Products](/combination-products)\n - [Regulatory Information](/regulatory-information)\n - [Safety](/safety)\n - [Emergency Preparedness](/emergency-preparedness-and-response)\n - [International Programs](/international-programs)\n - [News and Events](/news-events)\n - [Training and Continuing Education](/training-and-continuing-education)\n - [Inspections and Compliance](/inspections-compliance-enforcement-and-criminal-investigations)\n - [Science and Research](/science-research)\n\n\n\n\n\n\n\n## Information For\n\n\n - [Consumers](/consumers)\n - [Patients](/patients)\n - [Industry](/industry)\n - [Health Professionals](/health-professionals)\n - [Federal, State and Local Officials](/federal-state-local-tribal-and-territorial-officials)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Home](/)\n - [News & Events](/news-events)\n - [FDA Newsroom](/news-events/fda-newsroom)\n - [Press Announcements](/news-events/fda-newsroom/press-announcements)\n - FDA Approves First Drug to Treat Alexander Disease\n\n\n - [Press Announcements](/news-events/fda-newsroom/press-announcements)\n\n\n\n\n\n\n\n [In this section](#section-nav)\n\n\n - [Press Announcements](/news-events/fda-newsroom/press-announcements)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\nFDA News Release\n\n\n\n# FDA Approves First Drug to Treat Alexander Disease\n\n\n\nNew therapy is the first to directly target the underlying cause of this rare neurological disease\n\n\n\n\n\n\n\n\n\n\n\n\n\n[** More Press Announcements](/news-events/newsroom/press-announcements)\n\n\n\n\n -\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n For Immediate Release: September 03, 2026\n\n\n\n\n\n\n\nThe U.S. Food and Drug Administration has approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in pediatric and adult patients. Zanvastro is the first FDA-approved treatment for Alexander disease and the first therapy to directly target the protein buildup that drives the disease.\n\n“For patients with Alexander disease and their families, there have been no approved treatment options — only supportive care while the disease progresses,” **said Emily Freilich, M.D., Director of the Division of Neurology I in the FDA’s Center for Drug Evaluation and Research**. “Today’s approval is a landmark moment for this community, offering the first therapy that addresses the underlying cause of this rare and serious disease.”\n\nAlexander disease is a rare, progressive neurological disorder caused by mutations in the gene that produces glial fibrillary acidic protein (GFAP). When this protein is abnormal, it accumulates in the brain’s supportive cells, causing damage to the nervous system over time. The disease affects less than 1 in a million people and can cause a wide range of serious and potentially life-threatening problems, including seizures, loss of developmental milestones, difficulty walking, muscle weakness, and increased pressure in the brain.\n\nZanvastro is an antisense oligonucleotide — a type of medicine that works by reducing the production of the abnormal GFAP protein before it can accumulate and cause further damage. It is administered as an injection into the spinal canal every three months by a trained healthcare professional.\n\nThe efficacy and safety of Zanvastro were evaluated in a multicenter, randomized, controlled clinical study ([NCT04849741](https://clinicaltrials.gov/study/NCT04849741?term=%5BREDACTED%5D&viewType=%5BREDACTED%5D&rank=%5BREDACTED%5D)) enrolling 49 pediatric and adult patients with Alexander disease 2 years of age and older, and in an open-label substudy of 4 patients less than 2 years of age. Because Alexander disease is so rare and can affect patients across a wide range of ages, the FDA evaluated the available evidence in support of an indication covering patients from infancy through adulthood.\n\nIn patients aged 5 years and older who had measurable difficulties with walking at baseline, those treated with Zanvastro showed significantly better walking speed at 61 weeks compared to those who received no treatment. In children aged 2 to 4 years, where walking speed is not a reliable measure of progress, a broader assessment of motor skills — including standing, walking, running, and jumping — was used instead. Children treated with Zanvastro showed improvement on this measure while those in the control group declined.\n\nFor patients under 2 years of age, direct clinical trial data were limited by the rarity of the disease and lack of concurrent control. Pharmacokinetic modeling confirmed that drug levels in this age group are expected to be similar to those seen in older children at the same dose. This observation was also supported by safety data from four patients under 2 years of age treated in the study, as well as the safety data observed in the older pediatric patients. This approach supported extending the indication to the youngest patients while maintaining the evidentiary standards required to support approval.\n\nThe most common side effects of Zanvastro are vomiting, back pain, cough, headache, and post-lumbar puncture syndrome. Patients and caregivers should inform their healthcare provider if symptoms consistent with meningitis develop, as aseptic meningitis has been reported in patients treated with Zanvastro. Patients and providers should carefully review the full prescribing information when making treatment decisions.\n\nZanvastro received [Orphan Drug](/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products), [Fast Track](/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track), [Breakthrough Therapy](/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy), and [Rare Pediatric Disease and Priority Review Voucher](/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs) designations, reflecting the serious and rare nature of Alexander disease, and FDA’s commitment to rare disease drug development.\n\nThe approval of Zanvastro for the treatment of Alexander disease was granted to Ionis Pharmaceuticals, Inc.\n\n---\n\n\n\n**Media:**\n[FDA Request for Comment](https://www.hhs.gov/request-for-comment-form/index.html?Agency=%5BREDACTED%5D)\n202-690-6343\n\n\n\n\n\n\n\n**Consumer:**\n888-INFO-FDA\n\n\n\n\n\n\n\n\n\n###\n\n\n\n\n\n\n\nBoilerplate\n\n\n\nThe FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.\n\n\n\n\n\n\n\n---\n\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/03/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n -\n\n\n\n## Follow FDA\n\n\n - [Follow @US_FDA on X](https://x.com/FDA)\n - [Follow FDA on Facebook](https://www.facebook.com/FDA)\n - [Follow @FDAMedia on X](https://x.com/FDAMedia)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact FDA](/about-fda/contact-fda) [Follow FDA on Facebook](https://www.facebook.com/FDA) [Follow FDA on X](https://x.com/FDA) [Follow FDA on Instagram](https://instagram.com/FDA)\n [Follow FDA on LinkedIn](https://www.linkedin.com/company/fda/) [View FDA videos on YouTube](https://youtube.com/@US_FDA) [Subscribe to FDA RSS feeds](/about-fda/contact-fda/subscribe-podcasts-and-news-feeds)\n\n [](/)\n\n Contact Number 1-888-INFO-FDA (1-888-463-6332)\n\n\n\n\n\n Back to Top",
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"content": "FDA grants accelerated approval to camizestrant with a CDK4/6 inhibitor for ESR1-Mutated HR-positive, HER2-negative locally advanced or metastatic breast cancer | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site.\n\n\n\n\n\n\n\n \n\n\n\n**The site is secure.**\n The **https://** ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n [](/)\n\n\n\n\n - Search\n - Menu\n\n\n\n\n\n\n\n Search FDA Submit search\n\n\n\n\n\n\n\n\n\n\n\n\n\n## Featured\n\n\n - [Report a Product Problem](https://contact.fda.gov/report-problem?utm_medium=%5BREDACTED%5D&utm_source=%5BREDACTED%5D)\n - [Contact FDA](/about-fda/contact-fda)\n - [FDA Guidance Documents](/regulatory-information/search-fda-guidance-documents)\n - [Recalls, Market Withdrawals and Safety Alerts](/safety/recalls-market-withdrawals-safety-alerts)\n - [Press Announcements](/news-events/newsroom/press-announcements)\n - [Warning Letters](/inspections-compliance-enforcement-and-criminal-investigations/compliance-actions-and-activities/warning-letters)\n - [Advisory Committees](/advisory-committees)\n - [En Español](/fda-en-espanol)\n\n\n\n\n\n\n\n\n\n\n\n## Products\n\n\n - [Food](/food)\n - [Drugs](/drugs)\n - [Medical Devices](/medical-devices)\n - [Radiation-Emitting Products](/radiation-emitting-products)\n - [Vaccines, Blood, and Biologics](/vaccines-blood-biologics)\n - [Animal and Veterinary](/animal-veterinary)\n - [Cosmetics](/cosmetics)\n - [Tobacco Products](/tobacco-products)\n\n\n\n\n\n\n\n## Topics\n\n\n - [About FDA](/about-fda)\n - [Combination Products](/combination-products)\n - [Regulatory Information](/regulatory-information)\n - [Safety](/safety)\n - [Emergency Preparedness](/emergency-preparedness-and-response)\n - [International Programs](/international-programs)\n - [News and Events](/news-events)\n - [Training and Continuing Education](/training-and-continuing-education)\n - [Inspections and Compliance](/inspections-compliance-enforcement-and-criminal-investigations)\n - [Science and Research](/science-research)\n\n\n\n\n\n\n\n## Information For\n\n\n - [Consumers](/consumers)\n - [Patients](/patients)\n - [Industry](/industry)\n - [Health Professionals](/health-professionals)\n - [Federal, State and Local Officials](/federal-state-local-tribal-and-territorial-officials)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Home](/)\n - [Drugs](/drugs)\n - [Development & Approval Process | Drugs](/drugs/development-approval-process-drugs)\n - [Drug Approvals and Databases](/drugs/development-approval-process-drugs/drug-approvals-and-databases)\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n - FDA grants accelerated approval to camizestrant with a CDK4/6 inhibitor for ESR1-Mutated HR-positive, HER2-negative locally advanced or metastatic breast cancer\n\n\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n\n\n\n\n\n\n\n [In this section](#section-nav)\n\n\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n\n\n\n\n - [Oncology (Cancer)/Hematologic Malignancies Approval Notifications](/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications)\n - [Ongoing | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/ongoing-cancer-accelerated-approvals)\n - [Verified Clinical Benefit | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/verified-clinical-benefit-cancer-accelerated-approvals)\n - [Withdrawn | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/withdrawn-cancer-accelerated-approvals)\n - [Other | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/other-cancer-accelerated-approvals)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n# FDA grants accelerated approval to camizestrant with a CDK4/6 inhibitor for ESR1-Mutated HR-positive, HER2-negative locally advanced or metastatic breast cancer\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n\n\n\n\n - [Oncology (Cancer)/Hematologic Malignancies Approval Notifications](/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications)\n - [Ongoing | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/ongoing-cancer-accelerated-approvals)\n - [Verified Clinical Benefit | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/verified-clinical-benefit-cancer-accelerated-approvals)\n - [Withdrawn | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/withdrawn-cancer-accelerated-approvals)\n - [Other | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/other-cancer-accelerated-approvals)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\nOn September 4, 2026, the Food and Drug Administration granted accelerated approval to camizestrant (Etcamah, AstraZeneca), an estrogen receptor antagonist, in combination with a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib) for adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer upon detection of estrogen receptor-1 (*ESR1)* mutation during aromatase inhibitor and CDK4/6 inhibitor therapy, based on an FDA-authorized test.\n\nFDA also approved the Guardant360 CDx assay as a companion diagnostic device to identify patients with breast cancer with *ESR1* mutations for treatment with camizestrant.\n\nFull prescribing information for Etcamah will be posted on [Drugs@FDA](https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm).\n\n## Efficacy and Safety\n\nEfficacy was evaluated in SERENA-6 (NCT04964934), a randomized, double-blind, placebo-controlled, multicenter trial to assess switching to camizestrant in combination with a CDK4/6 inhibitor versus continuing an aromatase inhibitor (AI) in combination with a CDK4/6 inhibitor in adult patients with ER-positive, HER2-negative locally advanced or metastatic breast cancer with detectable *ESR1* mutations. The *ESR1* mutation status was assessed by blood circulating tumor deoxyribonucleic acid (ctDNA) testing using the Guardant360 CDx assay during first-line treatment with an AI and a CDK4/6 inhibitor. All patients were required to be currently receiving treatment of an AI in combination with a CDK4/6 inhibitor for ≥ 6 months as the initial endocrine based treatment with no evidence of disease progression as per investigator assessment. A total of 315 patients were randomized (1:1) to receive either camizestrant once daily in combination with a CDK4/6 inhibitor or AI (anastrozole or letrozole) orally once daily in combination with a CDK4/6 inhibitor.\n\nThe primary efficacy outcome measure was investigator-assessed progression-free survival (PFS) per RECIST v1.1. An additional efficacy outcome measure included overall survival (OS). Median PFS was 16 months (95% CI: 12.7, 18.2) in the camizestrant and CDK4/6 inhibitor arm and 9.2 months (95% CI: 7.2, 9.5) in the AI and CDK4/6 inhibitor arm (Hazard ratio 0.44 [95% CI: 0.31, 0.60]; p-value < 0.00001). At the time of the PFS analysis, OS data were not mature.\n\nThe prescribing information includes a boxed warning for the risk of arrhythmia due to QTc interval prolongation when concomitantly used with QTc interval prolonging drugs, as well as warnings and precautions for bradycardia and embryo-fetal toxicity.\n\n## Recommended Dosage\n\nThe recommended camizestrant dose is 75 mg orally once daily, with or without food, until disease progression or unacceptable toxicity. Administer camizestrant in combination with a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib). Continue the CDK4/6 inhibitor at the same dosage as when the *ESR1* mutation was detected. Refer to the prescribing information of the CDK4/6 inhibitor for additional dosing information.\n\nCamizestrant in combination with a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib) is being granted accelerated approval based on PFS measured from detection of *ESR1* mutation. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). For more information on ongoing accelerated approvals in oncology, refer to [Project Confirm](/about-fda/oncology-center-excellence/project-confirm).\n\nThis review was conducted under [Project Orbis](/about-fda/oncology-center-excellence/project-orbis), an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), the Brazilian Health Regulatory Agency (ANVISA), Health Canada, Singapore’s Health Sciences Authority (HSA), and Switzerland’s Swissmedic. The application reviews may be ongoing at the other regulatory agencies.\n\nThis application was discussed at an [Oncology Drugs Advisory Committee meeting](/advisory-committees/human-drug-advisory-committees/oncologic-drugs-advisory-committee). This review used the [Assessment Aid](/about-fda/oncology-center-excellence/assessment-aid), a voluntary submission from the applicant to facilitate the FDA’s assessment.\n\nThis application was granted standard review. Camizestrant received breakthrough therapy designation. A description of FDA expedited programs is in the [Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics](/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics).\n\nHealthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s [MedWatch Reporting System](https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) or by calling 1-800-FDA-1088.\n\nFor assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s [Project Facilitate](/about-fda/oncology-center-excellence/project-facilitate) at 240-402-0004 or email [OncProjectFacilitate@fda.hhs.gov](mailto:OncProjectFacilitate@fda.hhs.gov).\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/04/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact FDA](/about-fda/contact-fda) [Follow FDA on Facebook](https://www.facebook.com/FDA) [Follow FDA on X](https://x.com/FDA) [Follow FDA on Instagram](https://instagram.com/FDA)\n [Follow FDA on LinkedIn](https://www.linkedin.com/company/fda/) [View FDA videos on YouTube](https://youtube.com/@US_FDA) [Subscribe to FDA RSS feeds](/about-fda/contact-fda/subscribe-podcasts-and-news-feeds)\n\n [](/)\n\n Contact Number 1-888-INFO-FDA (1-888-463-6332)\n\n\n\n\n\n Back to Top",
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"content": "FDA grants accelerated approval to sevabertinib for locally advanced or metastatic non-squamous non-small cell lung cancer | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site.\n\n\n\n\n\n\n\n \n\n\n\n**The site is secure.**\n The **https://** ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n [](/)\n\n\n\n\n - Search\n - Menu\n\n\n\n\n\n\n\n Search FDA Submit search\n\n\n\n\n\n\n\n\n\n\n\n\n\n## Featured\n\n\n - [Report a Product Problem](https://contact.fda.gov/report-problem?utm_medium=%5BREDACTED%5D&utm_source=%5BREDACTED%5D)\n - [Contact FDA](/about-fda/contact-fda)\n - [FDA Guidance Documents](/regulatory-information/search-fda-guidance-documents)\n - [Recalls, Market Withdrawals and Safety Alerts](/safety/recalls-market-withdrawals-safety-alerts)\n - [Press Announcements](/news-events/newsroom/press-announcements)\n - [Warning Letters](/inspections-compliance-enforcement-and-criminal-investigations/compliance-actions-and-activities/warning-letters)\n - [Advisory Committees](/advisory-committees)\n - [En Español](/fda-en-espanol)\n\n\n\n\n\n\n\n\n\n\n\n## Products\n\n\n - [Food](/food)\n - [Drugs](/drugs)\n - [Medical Devices](/medical-devices)\n - [Radiation-Emitting Products](/radiation-emitting-products)\n - [Vaccines, Blood, and Biologics](/vaccines-blood-biologics)\n - [Animal and Veterinary](/animal-veterinary)\n - [Cosmetics](/cosmetics)\n - [Tobacco Products](/tobacco-products)\n\n\n\n\n\n\n\n## Topics\n\n\n - [About FDA](/about-fda)\n - [Combination Products](/combination-products)\n - [Regulatory Information](/regulatory-information)\n - [Safety](/safety)\n - [Emergency Preparedness](/emergency-preparedness-and-response)\n - [International Programs](/international-programs)\n - [News and Events](/news-events)\n - [Training and Continuing Education](/training-and-continuing-education)\n - [Inspections and Compliance](/inspections-compliance-enforcement-and-criminal-investigations)\n - [Science and Research](/science-research)\n\n\n\n\n\n\n\n## Information For\n\n\n - [Consumers](/consumers)\n - [Patients](/patients)\n - [Industry](/industry)\n - [Health Professionals](/health-professionals)\n - [Federal, State and Local Officials](/federal-state-local-tribal-and-territorial-officials)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Home](/)\n - [Drugs](/drugs)\n - [Development & Approval Process | Drugs](/drugs/development-approval-process-drugs)\n - [Drug Approvals and Databases](/drugs/development-approval-process-drugs/drug-approvals-and-databases)\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n - FDA grants accelerated approval to sevabertinib for locally advanced or metastatic non-squamous non-small cell lung cancer\n\n\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n\n\n\n\n\n\n\n [In this section](#section-nav)\n\n\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n\n\n\n\n - [Oncology (Cancer)/Hematologic Malignancies Approval Notifications](/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications)\n - [Ongoing | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/ongoing-cancer-accelerated-approvals)\n - [Verified Clinical Benefit | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/verified-clinical-benefit-cancer-accelerated-approvals)\n - [Withdrawn | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/withdrawn-cancer-accelerated-approvals)\n - [Other | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/other-cancer-accelerated-approvals)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n# FDA grants accelerated approval to sevabertinib for locally advanced or metastatic non-squamous non-small cell lung cancer\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [Resources for Information | Approved Drugs](/drugs/drug-approvals-and-databases/resources-information-approved-drugs)\n\n\n\n\n - [Oncology (Cancer)/Hematologic Malignancies Approval Notifications](/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications)\n - [Ongoing | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/ongoing-cancer-accelerated-approvals)\n - [Verified Clinical Benefit | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/verified-clinical-benefit-cancer-accelerated-approvals)\n - [Withdrawn | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/withdrawn-cancer-accelerated-approvals)\n - [Other | Cancer Accelerated Approvals](/drugs/resources-information-approved-drugs/other-cancer-accelerated-approvals)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\nOn September 9, 2026, the Food and Drug Administration granted accelerated approval to sevabertinib (Hyrnuo, Bayer Healthcare Pharmaceuticals Inc.), a kinase inhibitor, for adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-authorized test.\n\nThis approval expands the existing indication for sevabertinib, which was previously granted accelerated approval for adult patients with locally advanced or metastatic non-squamous NSCLC with HER2 (ERBB2) TKD activating mutations who had received prior systemic therapy.\n\nFull prescribing information for Hyrnuo will be posted on [Drugs@FDA](https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm).\n\n## Efficacy and Safety\n\nEfficacy was evaluated in patients with locally advanced or metastatic, non-squamous NSCLC with HER2 (ERBB2) TKD activating mutations who received sevabertinib in SOHO-01 (NCT05099172), an open-label, single-arm, multicenter, multi-cohort clinical trial. HER2 (ERBB2) activating mutations were determined in tumor tissue or plasma by local laboratories prior to enrollment.\n\nThe major efficacy outcome measures were confirmed objective response rate (ORR) and duration of response (DOR) as assessed by blinded independent central review (BICR) using RECIST v1.1. Among 69 patients with locally advanced or metastatic NSCLC with HER2 (ERBB2) TKD activating mutations who had not received prior systemic therapy, the ORR was 75% (95% CI: 64, 85); 73% of responding patients had a DOR of ≥6 months and 38% had a DOR of ≥12 months.\n\nThe prescribing information includes warnings and precautions for diarrhea, hepatotoxicity, interstitial lung disease (ILD)/pneumonitis, left ventricular dysfunction, ocular toxicity, pancreatic enzyme elevation, and embryo-fetal toxicity.\n\n## Recommended Dosage\n\nThe recommended sevabertinib dose is 20 mg orally twice daily with food, until disease progression or unacceptable toxicity.\n\nThis review was conducted under [Project Orbis](/about-fda/oncology-center-excellence/project-orbis), an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). The application reviews are ongoing at the other regulatory agencies.\n\nThis review used the [Assessment Aid](/about-fda/oncology-center-excellence/assessment-aid), a voluntary submission from the applicant to facilitate the FDA’s assessment.\n\nThis application was granted priority review. Sevabertinib received breakthrough therapy designation and orphan drug designation. A description of FDA expedited programs is in the [Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics](/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics).\n\nHealthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s [MedWatch Reporting System](https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) or by calling 1-800-FDA-1088.\n\nFor assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s [Project Facilitate](/about-fda/oncology-center-excellence/project-facilitate) at 240-402-0004 or email [OncProjectFacilitate@fda.hhs.gov](mailto:OncProjectFacilitate@fda.hhs.gov).\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n09/09/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact FDA](/about-fda/contact-fda) [Follow FDA on Facebook](https://www.facebook.com/FDA) [Follow FDA on X](https://x.com/FDA) [Follow FDA on Instagram](https://instagram.com/FDA)\n [Follow FDA on LinkedIn](https://www.linkedin.com/company/fda/) [View FDA videos on YouTube](https://youtube.com/@US_FDA) [Subscribe to FDA RSS feeds](/about-fda/contact-fda/subscribe-podcasts-and-news-feeds)\n\n [](/)\n\n Contact Number 1-888-INFO-FDA (1-888-463-6332)\n\n\n\n\n\n Back to Top",
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"description": "On September 9, 2026, the Food and Drug Administration granted accelerated approval to sevabertinib (Hyrnuo, Bayer Healthcare Pharmaceuticals Inc.), a kinase inhibitor, for adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB",
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"content": "FDA Approves First Drug of Its Kind for Polycythemia Vera, a Rare Blood Disorder | FDA\n\n\n - [Skip to main content](#main-content)\n - [Skip to FDA Search](#search-form)\n - [Skip to in this section menu](#section-nav)\n - [Skip to footer links](#footer)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n  An official website of the United States government Here’s how you know\n\n\n\n\n\n \n\n\n\n**The .gov means it’s official.**\nFederal government websites often end in .gov or .mil. 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The excess cells can thicken the blood and increase the risk of cardiovascular issues, such as blood clots, stroke and heart attack.\n\nMimrylo offers a new treatment approach for patients whose disease has not been adequately controlled with existing therapies. It is the first approved treatment for polycythemia vera that mimics hepcidin, a hormone that naturally regulates iron in the body. By limiting the iron available to make new red blood cells, Mimrylo helps reduce their overproduction and keep red blood cell levels under control. This novel approach offers a new option for patients who have not achieved adequate control with existing therapies.\n\n“People living with polycythemia vera have long faced the challenge of managing a chronic blood disorder with frequent blood draws to help address the consequences of the red blood cell overproduction,” **said Tanya Wroblewski, M.D., Director of the Division of Nonmalignant Hematology within the FDA’s Center for Drug Evaluation and Research**. “Today's approval of Mimrylo offers a new, first-in-class option that has the potential to meaningfully reduce patient burden.\"\n\nA key goal of treatment for polycythemia vera is keeping the proportion of red blood cells in the blood (hematocrit) below 45% to reduce cardiovascular risks. This often requires phlebotomy, a procedure that removes blood from a vein to lower the number of red blood cells in the body. However, some patients continue to need frequent phlebotomies despite treatment, creating an ongoing burden.\n\nThe safety and efficacy of Mimrylo were evaluated in the [VERIFY](https://clinicaltrials.gov/study/NCT05210790?id=%5BREDACTED%5D&viewType=%5BREDACTED%5D&rank=%5BREDACTED%5D) trial, a multicenter, randomized, double-blind, placebo-controlled phase 3 study of 293 adults with polycythemia vera who required frequent phlebotomies despite ongoing standard-of-care therapy. Patients were randomized 1:1 to receive either Mimrylo or placebo over 32 weeks. Mimrylo treatment started at 19 mg, administered subcutaneously (under the skin) once weekly, and was titrated to maintain hematocrit levels below 45%.\n\nEfficacy was measured by the proportion of patients who did not meet criteria for phlebotomy between weeks 20 and 32 of the study. Overall, 76.9% of patients on Mimrylo required no phlebotomies during the 32-week period compared to 32.9% on placebo. The most common adverse reactions were injection site reactions and anemia.\n\nMimrylo received [priority review](/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review). The approval was granted to Takeda Pharmaceuticals America, Inc.\n\n\n\n###\n\n\n\n\n\n\n\nBoilerplate\n\n\n\nThe FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.\n\n\n\n\n\n\n\n---\n\n\n\n\n\n\n\n\n\n -\n\n\n\n## Content current as of:\n\n\n\n08/28/2026\n\n\n\n\n -\n\n\n\n## Regulated Product(s)\n\n\n - Drugs\n\n\n\n\n -\n\n\n\n## Follow FDA\n\n\n - [Follow @US_FDA on X](https://x.com/FDA)\n - [Follow FDA on Facebook](https://www.facebook.com/FDA)\n - [Follow @FDAMedia on X](https://x.com/FDAMedia)\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n Feedback\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n - [FDA Archive](/about-fda/about-website/fdagov-archive)\n - [About FDA](/about-fda)\n - [Accessibility](/about-fda/about-website/internet-accessibility)\n\n\n\n\n\n\n - [Visitor Information](/about-fda/visitor-information)\n - [Website Policies / Privacy](/about-fda/about-website/website-policies)\n - [No FEAR Act](/about-fda/jobs-and-training-fda/no-fear-act)\n - [Vulnerability Disclosure Policy](https://www.hhs.gov/vulnerability-disclosure-policy/index.html)\n\n\n\n\n\n\n - [FOIA](/regulatory-information/freedom-information)\n - [HHS.gov](https://www.hhs.gov/)\n - [USA.gov](https://www.usa.gov/)\n\n\n\n\n\n\n\n\n\n\n\n [Contact FDA](/about-fda/contact-fda) [Follow FDA on Facebook](https://www.facebook.com/FDA) [Follow FDA on X](https://x.com/FDA) [Follow FDA on Instagram](https://instagram.com/FDA)\n [Follow FDA on LinkedIn](https://www.linkedin.com/company/fda/) [View FDA videos on YouTube](https://youtube.com/@US_FDA) [Subscribe to FDA RSS feeds](/about-fda/contact-fda/subscribe-podcasts-and-news-feeds)\n\n [](/)\n\n Contact Number 1-888-INFO-FDA (1-888-463-6332)\n\n\n\n\n\n Back to Top",
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"message": "Four novel approvals in the Aug 28 – Sep 11, 2026 window, per FDA's drug news pages:\n\n| Drug (ingredient) | Indication | Sponsor | Approval | Key label limits |\n|---|---|---|---|---|\n| [Isembyld (apitegromab-mstn)](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-therapy-target-muscle-loss-spinal-muscular-atrophy) | Spinal muscular atrophy, adults and children 2+ already on an SMN2-targeted treatment | Scholar Rock | 9/11/2026 | Add-on only, not monotherapy. Increased risk of fractures, including serious fractures. May cause fetal harm and may affect reproductive function. |\n| [Etcamah (camizestrant)](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-camizestrant-cdk46-inhibitor-esr1-mutated-hr-positive-her2-negative) | HR+/HER2- advanced or metastatic breast cancer, with a CDK4/6 inhibitor, on ESR1 mutation detected during AI + CDK4/6 therapy | AstraZeneca | 9/4/2026 | Accelerated approval on PFS; continued approval may require a confirmatory trial. Boxed warning for QTc prolongation/arrhythmia with other QTc-prolonging drugs. Bradycardia, embryo-fetal toxicity. Requires Guardant360 CDx companion test. |\n| [Zanvastro (zilganersen)](https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-alexander-disease) | Alexander disease, pediatric and adult | Ionis Pharmaceuticals | 9/3/2026 | Intrathecal injection every 3 months by a trained professional. Aseptic meningitis reported. Under-2 dosing rests on PK modeling plus 4 treated infants, not controlled data. |\n| [Mimrylo (rusfertide)](https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-its-kind-polycythemia-vera-rare-blood-disorder) | Erythrocytosis in adults with polycythemia vera | Takeda Pharmaceuticals America | 8/28/2026 | Weekly subcutaneous, titrated to keep hematocrit under 45%. Studied in patients still needing phlebotomy despite standard therapy. Injection site reactions and anemia most common. |\n\nAlso announced in the window but not novel agents: [sevabertinib (Hyrnuo, Bayer)](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sevabertinib-locally-advanced-or-metastatic-non-squamous-non-small) got expanded accelerated approval 9/9 to first-line HER2 TKD-mutant non-squamous NSCLC, plus new indications for Joenja (pediatric APDS, 9/11), Besremi (essential thrombocythemia, 8/31) and Stelara (pediatric ulcerative colitis, 8/28)."
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}| Drug (ingredient) | Indication | Sponsor | Approval | Key label limits |
|---|---|---|---|---|
| Isembyld (apitegromab-mstn) | Spinal muscular atrophy, adults and children 2+ already on an SMN2-targeted treatment | Scholar Rock | 9/11/2026 | Add-on only, not monotherapy. Increased risk of fractures, including serious fractures. May cause fetal harm and may affect reproductive function. |
| Etcamah (camizestrant) | HR+/HER2- advanced or metastatic breast cancer, with a CDK4/6 inhibitor, on ESR1 mutation detected during AI + CDK4/6 therapy | AstraZeneca | 9/4/2026 | Accelerated approval on PFS; continued approval may require a confirmatory trial. Boxed warning for QTc prolongation/arrhythmia with other QTc-prolonging drugs. Bradycardia, embryo-fetal toxicity. Requires Guardant360 CDx companion test. |
| Zanvastro (zilganersen) | Alexander disease, pediatric and adult | Ionis Pharmaceuticals | 9/3/2026 | Intrathecal injection every 3 months by a trained professional. Aseptic meningitis reported. Under-2 dosing rests on PK modeling plus 4 treated infants, not controlled data. |
| Mimrylo (rusfertide) | Erythrocytosis in adults with polycythemia vera | Takeda Pharmaceuticals America | 8/28/2026 | Weekly subcutaneous, titrated to keep hematocrit under 45%. Studied in patients still needing phlebotomy despite standard therapy. Injection site reactions and anemia most common. |
### FDA novel drug approvals, Aug 28–Sep 11, 2026 · FDA news feed | Drug (ingredient) | Indication | Sponsor | Approval | Key label limits | |---|---|---|---|---| | Isembyld (apitegromab-mstn) | Spinal muscular atrophy, adults and children 2+ already on an SMN2-targeted treatment | Scholar Rock | 9/11/2026 | Add-on only, not monotherapy. Increased risk of fractures, including serious fractures. May cause fetal harm and may affect reproductive function. | | Etcamah (camizestrant) | HR+/HER2- advanced or metastatic breast cancer, with a CDK4/6 inhibitor, on ESR1 mutation detected during AI + CDK4/6 therapy | AstraZeneca | 9/4/2026 | Accelerated approval on PFS; continued approval may require a confirmatory trial. Boxed warning for QTc prolongation/arrhythmia with other QTc-prolonging drugs. Bradycardia, embryo-fetal toxicity. Requires Guardant360 CDx companion test. | | Zanvastro (zilganersen) | Alexander disease, pediatric and adult | Ionis Pharmaceuticals | 9/3/2026 | Intrathecal injection every 3 months by a trained professional. Aseptic meningitis reported. Under-2 dosing rests on PK modeling plus 4 treated infants, not controlled data. | | Mimrylo (rusfertide) | Erythrocytosis in adults with polycythemia vera | Takeda Pharmaceuticals America | 8/28/2026 | Weekly subcutaneous, titrated to keep hematocrit under 45%. Studied in patients still needing phlebotomy despite standard therapy. Injection site reactions and anemia most common. |