Treats spinal muscular atrophy (SMA). Sold as Isembyld. Every FDA application for this active ingredient.
As of Nov 15, 2026, FDA lists 1 application for Apitegromab-Mstn (sold as Isembyld), first approved Sep 11, 2026, with 0 new uses approved and 1 published complete response letter.
| Phase 2 Study Evaluating Apitegromab for the Treatment of FSHDFacioscapulohumeral Muscular Dystrophy, FSHD · Scholar Rock, Inc. | Phase 2 | Recruiting | Feb 27, 2026 |
| A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophySpinal Muscular Atrophy, SMA · Scholar Rock, Inc. | Phase 2 | Recruiting | Jul 2, 2025 |
| Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabSpinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 · Scholar Rock, Inc. | Phase 3 | Active, not recruiting | Nov 25, 2022 |
| Approved | Application | Sponsor | Supplements | Marketing |
|---|---|---|---|---|
| Sep 11, 2026 | IsembyldBLA 761463 · New molecular entity | Scholar Rock Inc | 0 | Marketed |
| Letter | Product | Now |
|---|---|---|
| Sep 22, 2025 | BLA 761463Facility Inspections, Postmarketing Requirements Under 505(0)(3) | Unapproved |
Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA
