USFDAMay 27, 2026, 07:32 AM
BridgeBio BBP-418 NDA Accepted for Priority Review by FDA
AI Summary
BridgeBio Pharma announced the FDA has accepted its New Drug Application for oral BBP-418, granting it Priority Review for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The FDA set a PDUFA target action date of November 27, 2026, and is not currently planning an advisory committee meeting. If approved, BBP-418 would be the first and only therapy for LGMD2I/R9 and the first approved treatment for any form of LGMD, addressing a significant unmet medical need for approximately 7,000 individuals in the U.S. and Europe. The Phase 3 FORTIFY trial demonstrated strong efficacy, with treated individuals improving across all key endpoints.
Key Highlights
- FDA accepted New Drug Application (NDA) for oral BBP-418 with Priority Review for LGMD2I/R9.
- PDUFA target action date is set for November 27, 2026.
- BBP-418 could be the first and only therapy for LGMD2I/R9 and first for any form of LGMD.
- Phase 3 FORTIFY trial demonstrated strong efficacy, meeting all primary and secondary endpoints.
- FDA is not currently planning an advisory committee meeting for the application.
- Approximately 7,000 individuals in the U.S. and Europe live with LGMD2I/R9.
- BBP-418 has Orphan Drug, Fast Track, and Rare Pediatric Disease Designations.
- BridgeBio may qualify for a Priority Review Voucher if BBP-418 is approved.
Price Impact
More from BBIO