BridgeBio Files FDA NDA For BBP-418, Targets Breakthrough In Muscular Dystrophy Treatment

BridgeBio Pharma, Inc. has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration for BBP-418, an oral therapy targeting limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This marks a significant regulatory milestone for a condition that currently has no approved treatments.

The submission is supported by positive results from the Phase 3 FORTIFY trial, where BBP-418 met all primary and secondary endpoints at the 12-month interim analysis. If approved, the therapy could become the first treatment specifically for LGMD2I/R9 and potentially the first approved therapy for any form of limb-girdle muscular dystrophy, addressing a major unmet medical need.

“This NDA submission brings us one step closer to delivering the first approved therapy to individuals and families affected by LGMD2I/R9, a severe, progressive neuromuscular disease,” said Christine Siu, Chief Executive Officer of BridgeBio Neuromuscular. “This achievement not only reflects the strength of the data, but also our dedicated focus on addressing the urgency of a community that has long been waiting for meaningful treatment options. We are committed to working closely with the FDA to make this potential disease-modifying therapy available as quickly as possible.”

BBP-418 has received multiple regulatory designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease status from the FDA, as well as Orphan Drug designation from the European Medicines Agency. These designations may support an accelerated review process, and the company could also be eligible for a Priority Review Voucher upon approval.

BridgeBio is targeting a potential U.S. launch in late 2026 or early 2027, pending approval. In parallel, the company is working with European regulators to explore expedited pathways and plans to expand clinical development to include younger patients and other LGMD subtypes in future studies.

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