BridgeBio's BBP-418 Gets FDA Rare Pediatric Disease Designation For Treatment Of LGMD2I/R9

(RTTNews) - BridgeBio Pharma, Inc. (BBIO) announced on Tuesday that the FDA has granted Rare Pediatric Disease Designation for BBP-418 for the treatment of Limb-girdle Muscular Dystrophy Type 2I/R9 also known as LGMD2I/R9.

The company also mentioned that it has exceeded its interim analysis enrollment target and anticipates top-line interim data from its Phase 3 registrational study known as FORTIFY, in individuals with LGMD2I/R9 by 2025. The study involves a planned interim analysis at 12 months that focuses on evaluating glycosylated aDG as a surrogate endpoint to back Accelerated Approval.

If BBP-418 receives approval, BridgeBio could be eligible for a Priority Review Voucher based on obtaining the Rare Pediatric Disease Designation.