STAT+: Scholar Rock wins FDA approval for first drug to target SMA muscle loss
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.
Scholar Rock has secured FDA approval for Isembyld, the first therapy designed to address muscle loss associated with spinal muscle atrophy (SMA). The FDA greenlit the drug for use in both adults and children aged 2 and above who are already undergoing treatments aimed at boosting SMN2, a gene crucial for neurons responsible for movement.
In a pivotal late-stage clinical trial, Isembyld demonstrated promising results when paired with a medication that targets SMN2. Patients who received this combination showed significant improvement in motor skills over a year, while those in the placebo group experienced a decline. This development is a landmark moment for the SMA community, according to David Hallal, CEO of Scholar Rock.
He expressed his excitement in a press release, stating that after years of unsuccessful attempts to harness the potential of myostatin inhibition, Scholar Rock has achieved a groundbreaking therapeutic breakthrough.
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