Today, Scholar Rock shared that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational treatment for spinal muscular atrophy (SMA), with a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2026. This is a meaningful step forward in the path toward a potential new treatment option targeting the muscles for the SMA community.
What This Means
When the FDA accepts an application of this kind, it has determined that the submission is complete and ready for a full review. This review period is expected to take up to six months. While acceptance does not guarantee that the treatment will be approved, it confirms that the formal evaluation process is now underway. Scholar Rock has indicated that, if approved, it will work to make apitegromab available as quickly as possible.
How We Got Here
As we shared on March 31, 2026, Scholar Rock resubmitted its application after the FDA previously raised concerns related to a third-party manufacturing facility, concerns that were not about apitegromab's safety or effectiveness. Scholar Rock addressed those concerns in close coordination with the FDA and also added a second U.S.-based manufacturing facility to help ensure a reliable supply if the treatment is approved.
What's Next
Cure SMA will remain in close communication with Scholar Rock and will continue our advocacy to the FDA throughout the review process. We are committed to keeping the SMA community informed and will share updates as they become available.
Click here to read Scholar Rock’s community statement.
Click here to read our updated F.A.Q.s.
If you have questions, please reach out to us at [email protected].