FDA approves Scholar Rock's muscle-targeted SMA therapy Isembyld

The Food and Drug Administration has approved a new medication for spinal muscular atrophy (SMA). The drug, apitegromab marketed as Isembyld, is the first muscle-targeted therapy for the disease.

The approval allows Isembyld to be used alongside existing targeted treatments in adults and children aged two years and older. Clinical testing showed it improved motor function in patients already receiving standard therapies.

The drug carries an annual list price of about $310,000, with actual costs varying by patient weight and insurance coverage. Executives disclosed the pricing during a recent conference call.

SMA, a rare genetic disorder affecting up to 25,000 people in the U.S., has seen a dramatic shift in treatment options since the first therapies were approved in 2016, moving from solely supportive care to disease-modifying drugs.