BridgeBio Shifts Filing Plans After Phase 3 Success in Rare Disease Drug BBP-418

BridgeBio's experimental therapy BBP-418 (ribitol) for limb-girdle muscular dystrophy type 2I/R9 achieved significant success in its Phase 3 trial, meeting all primary and secondary interim endpoints including sustained and statistically significant improvements in the glycosylation of alpha-dystroglycan (αDG), a key biomarker for the disease3 5 9.

Following this Phase 3 success, BridgeBio is preparing to meet with the FDA and plans to file for regulatory approval in the first half of 2026. The company is considering seeking full approval, potentially as the first approved therapy for this rare muscular disease7 11 15.

The company’s management and analysts see the data as robust enough not only to support accelerated approval based on biomarker improvement but also to potentially justify a traditional full approval filing, which would mark a shift from typical rare disease drug filing strategies7 15.

Regulatory discussions in the coming months will determine if the data package is sufficient for full rather than accelerated approval; the FDA has previously considered αDG a suitable surrogate endpoint for accelerated review3 15.

BBP-418, if approved, would supplement BridgeBio’s expanding rare disease portfolio alongside its already-commercialized drug acoramidis (Attruby) for cardiomyopathy. The company is leveraging its strong cash position and commercial infrastructure to accelerate filings and launches for rare genetic disease drugs1 3.