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.”
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