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

Scholar Rock on Friday received Food and Drug Administration approval for the first-ever therapy that targets the loss of muscle in spinal muscle atrophy, raising hopes that patients with the rare neurological disorder might have a better chance of moving and walking independently. The FDA approved the drug, Isembyld, for use in 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. “Today’s FDA approval of Isembyld marks a defining moment for the SMA community,” said David Hallal, CEO of Scholar Rock, in a press release. “After decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough.” STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe