“With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.”
Today, Scholar Rock announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab, a muscle-targeted therapy designed and developed to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments. To note, the FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA.
The Company's BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.
Scholar Rock also remains on track to file a Marketing Authorisation Application to the European Medicines Agency in 1Q 2025.
Next Steps
Scholar Rock has requested Priority Review which, if granted, would shorten the FDA’s review time to six months from the date of filing acceptance. The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations to apitegromab for the treatment of SMA.
In August 2024, Cure SMA hosted a webinar for the community that reviewed the important steps that will follow positive results from clinical trials that could lead to regulatory filing and potentially next treatment approvals. Click here to see a recording.
Cure SMA will continue to keep the community updated on news as Scholar Rock's Biologics License Application works its way through the review process toward potential approval.