BridgeBio’s BBP-418 Gets FDA Priority Review, PDUFA Date Nov. 27, 2026
BBIO•FDA granted Priority Review for BBP-418 with a PDUFA action date of November 27, 2026, for treating LGMD2I/R9. Phase 3 FORTIFY results showed treated patients improved across all primary and secondary endpoints while placebo recipients declined, addressing an estimated 7,000 patients in the U.S. and Europe.
1. NDA Acceptance and Priority Review
The FDA accepted BridgeBio’s New Drug Application for BBP-418 and granted Priority Review with a PDUFA action date of November 27, 2026. The agency also indicated it is not planning an advisory committee meeting, potentially expediting the approval process.
2. Phase 3 FORTIFY Trial Outcomes
In the FORTIFY trial interim analysis at 12 months, BBP-418 met all primary and secondary endpoints, with treated participants showing improvement across every key measure while placebo recipients declined. These results highlight the drug’s potential to alter the progressive course of LGMD2I/R9.
3. First Potential LGMD Therapy and Market Size
If approved, BBP-418 would become the first therapy for individuals with LGMD2I/R9 and the first approved treatment for any form of limb-girdle muscular dystrophy. Approximately 7,000 people in the U.S. and Europe live with LGMD2I/R9 or related α-dystroglycanopathies, representing a significant unmet medical need.
4. Regulatory and Development Pathway
BBP-418 holds multiple designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease, positioning it for potential Priority Review Voucher eligibility. BridgeBio plans to pursue approval in Europe and initiate studies in pediatric patients and other LGMD genotypes in the near term.
