The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 27, 2026, and BridgeBio is poised to launch BBP-418 upon approval. The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.

"LGMD2I/R9 is a relentless and life-shortening disease. Patients progressively lose the ability to walk, face serious cardiovascular complications, and ultimately die from respiratory failure," said Christine Siu, Chief Executive Officer of BridgeBio Neuromuscular. "With today's acceptance of our NDA, we are one step closer to the potential FDA approval of a treatment that could potentially change the progression of this disease. The compelling data from FORTIFY give us confidence that BBP-418 can make a meaningful difference in how this disease progresses, and we will work with urgency to bring it to the patients and families who have been waiting."

In the Phase 3 FORTIFY trial, BBP-418 met all primary and secondary endpoints at the pre-specified 12-month interim analysis, showing treated individuals improving while placebo recipients declined across every key measure. These results were presented as a late-breaking oral presentation at the 2026 MDA Clinical and Scientific Conference, which are available here.

"For families living with LGMD2I/R9, every milestone reflects years of advocacy, resilience, and hope," said Kat Bryant Knudson, Founder and CEO of The Speak Foundation. "The FDA's acceptance of this application reflects not only scientific advancement, but a commitment to listening to and partnering with our community every step of the way. We are grateful for the continued commitment to patients and families who have waited far too long."

BridgeBio believes BBP-418 is positioned to become the first and only approved therapy for individuals living with LGMD2I/R9 addressing a significant unmet need in this disease and potentially representing the first approval of a therapy for any form of LGMD. Approximately 7,000 individuals currently live with LGMD2I/R9 and other addressable α-dystroglycanopathies in the U.S. and Europe. The Company is also engaging regulatory agencies to identify an expedited path to approval for BBP-418 in Europe.

BBP-418 has previously received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA). BBP-418 received Priority Review from the FDA, highlighting the potential for BBP-418 to address unmet need in LGMD2I/R9. Consistent with Rare Pediatric Designation from the FDA, if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher. The Company intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age and in LGMD2M/R13 and LGMD2U/R20 in the near future.