FDA approves Isembyld, first therapy to target muscle loss in SMA patients

Scholar Rock received FDA approval for Isembyld, the first drug designed to address muscle loss in spinal muscular atrophy (SMA).

The approval covers adults and children aged two years and older who are already on SMN2-targeting therapies, adding a myostatin-inhibiting approach to their treatment regimen.

In a late-stage trial, patients who received Isembyld together with an SMN2-targeting drug showed measurable improvements in motor skills after one year, while those on placebo experienced a decline; the difference was statistically significant.

CEO David Hallal called the decision a defining moment for the SMA community, noting that the success follows decades of attempts to harness myostatin inhibition.