Scholar Rock gains FDA approval for ISEMBYLD in SMA

Scholar Rock has received US Food and Drug Administration (FDA) approval for ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children aged two years and older who are receiving an SMN2-targeted treatment.

ISEMBYLD is a muscle-targeted therapy designed to act on the myostatin pathway, addressing the muscle component of SMA.
 
The approval makes it the first treatment of its kind for patients receiving an SMN2-targeted therapy.

The FDA decision was supported by results from the Phase 3 SAPPHIRE study, a randomised, double-blind and placebo-controlled trial. 

After one year, patients treated with ISEMBYLD and an SMN2-targeted treatment showed a 2.2-point improvement in the Hammersmith Functional Motor Scale-Expanded (HFMSE) compared with those receiving an SMN2-targeted treatment alone.

A 3-point or greater improvement in HFMSE was reported in 34.2% of patients receiving ISEMBYLD, compared with 13.5% in the placebo group.

Safety data include more than 500 people treated with apitegromab across clinical studies. 
Common adverse reactions in SAPPHIRE included upper respiratory tract infections, vomiting, cough, viral infections, headache, gastroenteritis, pharyngitis and hypersensitivity.

Scholar Rock has also launched patient support services covering insurance, financial assistance, treatment education and infusion arrangements. 

ISEMBYLD is expected to become available in the US shortly.