Scholar Rock gets first FDA nod for rare muscle-wasting disease
The approval follows late-stage trial data showing the drug improved motor function and could help patients with a rare disorder move independently.
- On Friday, The FDA approved Isembyld as the first muscle-targeted therapy for spinal muscular atrophy , designed to improve motor function in adults and children 2 years and older.
- Spinal muscular atrophy is a genetic disorder affecting roughly 10,000 people in the United States by damaging motor neurons; Isembyld blocks myostatin, a protein limiting muscle growth, for patients receiving SMN2-targeting SMA therapies.
- A late-stage clinical trial found the therapy significantly improved motor skills in young patients compared to placebo, with CEO David Hallal stating "Today's FDA approval of Isembyld marks a defining moment."
- Scholar Rock shares rose over 23% in after-market trading following the approval, while BMO Capital Markets analyst Evan Seigerman forecasts $2.1 billion in adjusted worldwide peak sales by 2035.
- Testing is also underway to use Isembyld for muscle preservation during weight loss, competing with more than a dozen firms as the drug enters a market dominated by Biogen, Novartis, and Roche.
11 Articles
11 Articles
Scholar Rock gets first FDA nod for rare muscle-wasting disease - Regional Media News
By Sneha S K and Christy Santhosh Sept 11 (Reuters) - The U.S. Food and Drug Administration approved Scholar Rock's therapy on Friday to treat a rare genetic condition that causes muscle weakness, marking the first regulatory approval for the company. The injectable treatment, sold [...]
Scholar Rock gets first FDA nod for rare muscle-wasting disease
By Sneha S K and Christy Santhosh Sept 11 (Reuters) - The U.S. Food and Drug Administration approved Scholar Rock's therapy on Friday to treat a rare genetic condition that causes muscle weakness, marking the first regulatory approval for the company...
FDA Approves First Muscle-Targeted Therapy for SMA: A Conversation with Dr. Se-Jin Lee
The FDA has approved Isembyld (apitegromab-mstn), the first therapy designed to target muscle health and regeneration in people living with spinal muscular atrophy (SMA). This milestone adds to the progress made by genetic therapies for SMA and provides families with a new option focused on improving muscle strength and daily function. To help families understand… The post FDA Approves First Muscle-Targeted Therapy for SMA: A Conversation with D…
FDA Approves Isembyld (apitegromab-mstn), the First and Only Muscle-Targeted Therapy for Spinal ...
New York, Sept. 11, 2026 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) marks another milestone for the neuromuscular community with the U.S. Food and Drug Administration (FDA) approval of Isembyld (apitegromab-mstn), a monthly infusion treatment for people living with…
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