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 assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. The FDA priority review designation conveys that the FDA has determined that if apitegromab is approved, it could offer significant improvement in the safety or effectiveness of treatment of the serious condition of SMA.
Apitegromab’s regulatory submissions are based on positive efficacy and safety data from the pivotal Phase 3 SAPPHIRE trial, for which the Company reported positive topline data in October 2024, as well as supportive data from the Phase 2 TOPAZ trial and long-term extension ONYX trial.
Additionally, Scholar Rock recently announced an Expanded Access Program (EAP) in the U.S. for apitegromab for the treatment of eligible patients with SMA. More information can be found about the program here.
Next Steps
In anticipation of potential regulatory approvals, Scholar Rock is planning for a U.S. commercial launch upon approval in 2025, with European launch anticipated in 2026.
Cure SMA will continue to keep the community updated on news as Scholar Rock's application works its way through the FDA priority review process toward potential regulatory approval.