BBP-418 is an orally administered small-molecule therapy in development for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The program has advanced to NDA submission in the United States based on Phase 3 interim data, but approval was still pending at the time of source publication. It is intended as a disease-modifying substrate supplementation approach targeting glycosylation defects associated with FKRP mutations.
BridgeBio shares rose 2.10% to $69.41 after Phase 3 FORTIFY interim data for oral BBP-418 in LGMD2I/R9 showed broad efficacy and placebo-matched safety. The company plans a U.S. FDA filing in H1 2026.
BridgeBio Pharma reported long term acoramidis data in transthyretin amyloid cardiomyopathy and submitted a US FDA NDA for BBP-418 in LGMD2I/R9. BBP-418 could become the first approved therapy for the disease if cleared.
BridgeBio Pharma will present interim analysis data from its Phase 3 FORTIFY trial of BBP-418 for limb-girdle muscular dystrophy at the 2026 MDA conference. The study has met efficacy endpoints, with additional presentations planned from academic collaborators. The company previously announced its Q4 and full year 2025 financial results release date.
BridgeBio Pharma announced positive Phase 3 topline results for infigratinib in children with achondroplasia, meeting primary and key secondary endpoints. The company plans regulatory submissions in the U.S. and Europe in the second half of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT04800874 |
Study of BBP-418 in Patients With LGMD2I |
ACTIVE_NOT_RECRUITING | PHASE2 |
