FDA Grants Priority Review BLA for Spinal Muscular Atrophy Drug, Apitegromab

MDA 2025: Apitegromab has potential to fill an SMA need, exec says

The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an add-on treatment for spinal muscular atrophy (SMA), with a decision expected around the start of fall. Scholar Rock, the company developing apitegromab, applied seeking FDA approval earlier this year. This week, the FDA granted priority review to the application and set Sept. 22 as its decision date, the company anno…

FDA Grants Priority Review BLA for Spinal Muscular Atrophy Drug, Apitegromab

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

FDA Grants Priority Review for Biologics License Application (BLA) for Apitegromab as a Treatment for Spinal Muscular Atrophy

Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment. The FDA will review the application under priority review and has assi…