Business
BridgeBio Pharma Surpasses Interim Analysis Enrollment Target and Receives U.S. FDA Rare Pediatric Disease Designation for BBP-418, a Potential Treatment for Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
- BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line results from the interim analysis expected in 2025 - Recent Type C interactions with U.S. Food and Drug Administration (FDA) focused on the validated glycosylated alpha-dystroglycan (αDG) bioassay and our interim analysis plans reinforce BridgeBio's belief that there is potential to pursue Accelerated Approval for BBP-418 - Rare Pediatric Disease Designation for BBP-418 highlights that LGMD2I/R9 is a rare disease with serious manifestations, which primarily impacts children, and if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher PALO ALTO, Calif., June 18, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, today 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 αDG 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. More information about the ongoing Phase 3 clinical trial of BBP-418 (NCT05775848) can be found here on clinicaltrials.gov. Multiple encouraging discussions with the FDA in 2024, focused on the validated glycosylated αDG bioassay and our interim analysis plans, continue to support the Company’s plan to pursue Accelerated Approval for BBP-418 based on the use of glycosylated αDG levels as a surrogate endpoint in LGMD2I/R9. Furthermore, the Agency indicated that the Company’s approach to measure glycosylated αDG levels via a novel, validated muscle tissue-based bioassay appears reasonable. A peer-reviewed manuscript ...