STAT+: Scholar Rock wins FDA approval for first drug to target SMA muscle loss

Scholar Rock on Friday received FDA approval for Isembyld, the first-ever therapy that targets the loss of muscle in spinal muscular atrophy (SMA), raising hopes that patients with the rare neurological disorder might have a better chance of moving and walking independently.

The FDA approved Isembyld for adults and children 2 years and older who are currently receiving SMA therapies that target SMN2, a key gene for neurons that control movement. A late-stage clinical trial found that, when combined with an SMN2-targeting drug, Isembyld caused young patients’ motor skills to improve after a year, while those in a placebo group declined—a difference that was statistically significant.

Scholar Rock CEO David Hallal said in a press release that the approval marks a defining moment for the SMA community, adding that after decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough.