Scholar Rock wins FDA approval for muscle-targeted SMA treatment

Scholar Rock ($SRRK) received FDA approval for ISEMBYLD, its apitegromab treatment for spinal muscular atrophy, in patients aged two and older receiving SMN2-targeted therapy. The company plans to begin shipping the medicine within days. The decision moves its muscle-targeted approach from clinical development into commercial use and also brings a rare pediatric disease priority review voucher.

The supporting SAPPHIRE study assessed motor function alongside existing treatment. In the specified 10 mg/kg group of patients aged two to 12, the difference versus placebo was 2.2 points on the HFMSE scale after one year. Safety information remains important: fractures occurred in 9% of treated patients versus 2% on placebo. Approval does not establish a cure or replace the accompanying SMN2-targeted treatment.

Sources:

• Scholar Rock — approval announcement, Business Wire