BridgeBio Pharma, Inc. (NASDAQ: BBIO) submitted a New Drug Application to the FDA for BBP-418, an oral treatment for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), according to a company statement.

The submission includes interim data from the Phase 3 FORTIFY trial, which met all pre-specified primary and secondary endpoints in its 12-month analysis. The trial demonstrated improvements in ambulation and pulmonary function with the treatment.

BridgeBio anticipates a U.S. launch in late 2026 or early 2027 following FDA approval. If approved, BBP-418 would be the first therapy specifically for LGMD2I/R9 and potentially the first approved treatment for any form of limb-girdle muscular dystrophy.

The drug has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA, along with Orphan Drug designation from the European Medicines Agency. The Fast Track designation may make the application eligible for Priority Review.

LGMD2I/R9 is a genetic disease caused by mutations in the FKRP gene that typically affects muscle function in the limbs and can progress to involve pulmonary and cardiac muscles. The condition affects mobility, breathing, and heart function in patients.

BridgeBio plans to initiate clinical studies of BBP-418 in patients under 12 years old with LGMD2I/R9 and in patients with LGMD2M/2U. The company is also engaging European regulatory agencies for approval pathways outside the United States.