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FDA Approves First Drug to Target Muscle Loss in Spinal Muscular Atrophy

Scholar Rock's Isembyld has won FDA approval as the first therapy aimed at muscle loss in spinal muscular atrophy, after a trial found it improved motor skills where a placebo group declined.

Step by step

  1. 1

    SMN2-targeting therapy already in use

  2. 2

    Isembyld added in late-stage trial

  3. 3

    Motor skills improved after one year

  4. 4

    Placebo group's motor skills declined

  5. 5

    FDA grants approval for ages 2+

Scholar Rock announced Friday that it has received U.S. Food and Drug Administration (FDA) approval for Isembyld, described as the first-ever therapy to target the loss of muscle in spinal muscular atrophy (SMA), a rare neurological disorder. The approval raises hopes that patients might have a better chance of moving and walking independently.

The FDA approved Isembyld for use in adults and children aged 2 and older who are already receiving SMA therapies that target SMN2, a gene important for the neurons that control movement. In a late-stage clinical trial, adding Isembyld to an SMN2-targeting drug led young patients' motor skills to improve after a year, while those in a declined -- a difference the company says was statistically significant.

"Today's FDA approval of Isembyld marks a defining moment for the SMA community," said David Hallal, chief executive of Scholar Rock, in a statement. "After decades of failed industry-wide efforts to unlock the potential of , Scholar Rock has delivered a therapeutic breakthrough."

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#FDA approval#spinal muscular atrophy#Scholar Rock#Isembyld#rare disease
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