FDA approves Scholar Rock drug for spinal muscular atrophy muscle loss

The short version

• The FDA approved Scholar Rock's Isembyld, the first treatment specifically targeting muscle loss associated with spinal muscular atrophy.[STAT]
• Approval applies to pediatric patients aged 2 and older as well as adults who are already on SMN2-targeting therapies.[STAT]
• A late-stage trial demonstrated statistically significant motor skill improvements over one year when paired with standard treatment.[STAT]
• Scholar Rock's leadership described the regulatory decision as a major breakthrough following decades of industry attempts.[STAT]

Key facts

• Scholar Rock received FDA approval for Isembyld as the first therapy targeting muscle loss in spinal muscular atrophy.[STAT]
• The clearance covers adults and children aged 2 years and older who currently take SMN2-targeting drugs.[STAT]
• Late-stage clinical trial data showed statistically significant motor skill gains after one year in young patients on combination therapy versus placebo.[STAT]

Sources

Outlet counts describe coverage, not independent confirmation. Reports may share a wire service or original source.

• STAT+: Scholar Rock wins FDA approval for first drug to target SMA muscle lossSTAT metered