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)

Core Insights - BridgeBio Pharma has exceeded its interim analysis enrollment target for the Phase 3 FORTIFY study of BBP-418, with top-line results expected in 2025 [1] - The FDA has engaged in discussions regarding the glycosylated alpha-dystroglycan bioassay, supporting the potential for Accelerated Approval of BBP-418 [3] - BBP-418 has received Rare Pediatric Disease Designation, indicating its significance for children affected by LGMD2I/R9, and may qualify BridgeBio for a Priority Review Voucher upon approval [4] Group 1: Study Details - The FORTIFY study is a randomized, double-blind, placebo-controlled Phase 3 trial assessing the safety and efficacy of BBP-418 for LGMD2I/R9, with an interim analysis planned at 12 months [2] - The primary endpoint at 36 months will be the North Star Assessment for limb-girdle muscular dystrophies, aimed at providing confirmatory clinical data [2] - Enrollment for the FORTIFY study is ongoing in the U.S., UK, Europe, and Australia [2] Group 2: FDA Interactions - Recent discussions with the FDA have focused on the validated glycosylated αDG bioassay and interim analysis plans, reinforcing the strategy for Accelerated Approval [3] - The FDA has indicated that the approach to measure glycosylated αDG levels through a validated muscle tissue-based bioassay is reasonable [3] - A peer-reviewed manuscript detailing the novel assay has been published, supporting the scientific basis for the study [3] Group 3: Disease Background - LGMD2I/R9 is a rare, monogenic autosomal recessive disease caused by mutations in the FKRP gene, leading to impaired glycosylation of αDG [5] - Clinical manifestations typically begin in late childhood, with significant progression leading to loss of mobility and potential cardiac involvement [5] - The disease has a high severity profile, with 25% of individuals experiencing loss of independent ambulation and 30% developing cardiomyopathy in adulthood [5] Group 4: Company Overview - BridgeBio Pharma is a commercial-stage biopharmaceutical company focused on developing transformative medicines for genetic diseases [6] - Founded in 2015, the company aims to leverage advances in genetic medicine to expedite patient access to new therapies [6]