BridgeBio Pharma, Inc. announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study (FORTIFY) in individuals with LGMD2I/R9 in 2025. FORTIFY is a randomized, double-blind, placebo-controlled Phase 3 study evaluating the safety and efficacy of BBP-418, an investigational oral therapy in development for the treatment of individuals living with LGMD2I/R9. The study includes a planned interim analysis at 12 months focused on assessing glycosylated aDG as a surrogate endpoint to support Accelerated Approval.

The primary endpoint to be evaluated at 36 months is the North Star Assessment (NSAD) for limb-girdle type muscular dystrophies and is designed to provide confirmatory clinical data. BridgeBio is continuing to enroll FORTIFY in the U.S., UK, Europe, and Australia. Multiple encouraging discussions with the FDA in 2024, focused on the validated glycosylated aDG bioassay and interim analysis plans, continue to support the Company?s plan to pursue Accelerated Approval for BBP-418 based on the use of glycosylated aDG levels as a surrogate endpoint in LGMD2I/R9.

Furthermore, the Agency indicated that the Company?s approach to measure glycosylated aDG levels via a novel, validated muscle tissue-based bioassay appears reasonable. The Company also announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) for BBP-418 in the treatment of LGMD2I/R9, recognizing the rarity of this disease is characterized by serious manifestations primarily affecting children. If BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher based on receipt of the RPDD.

A Priority Review Voucher can be applied to another therapy in the Company?s pipeline for a shorter timeline during the review process of a New Drug Application or can be sold and transferred to another company looking to receive priority review for one of its applications.