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# BridgeBio Plans NDA for Muscular Dystrophy Therapy
- URL: https://www.fdaweb.com/bridgebio-plans-nda-for-muscular-dystrophy-therapy/
- Published: 2025-10-27T12:00:00.000Z
- Updated: 2026-09-14T15:25:05.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5160106

BridgeBio Pharma plans to file an NDA next year after reporting positive data from Phase 3 FORTIFY trial evaluating BBP-418, an oral small-molecule therapy for limb-girdle muscular dystrophy type 2I/R9\. The progressive genetic muscular dystrophy is caused by FKRP mutations, which impair glycosylation of alpha-dystroglycan (αDG) and lead to loss of mobility, respiratory decline, and cardiac complications, according to the company. There are currently no FDA-approved therapies that modify the disease.

BridgeBio says the pivotal data showed:

- Restoration of αDG glycosylation: 1.8-fold improvement from baseline at 3 months, sustained at 12 months
- Muscle damage marker impact: 82% mean reduction in serum creatine kinase at 12 months
- Motor function improvement: 0.27 meters per second faster velocity versus placebo on the 100-meter timed test
- • Pulmonary function gains: Approximately 5% difference vs placebo in forced vital capacity at 12 months

Safety was described as well-tolerated, with no new concerns relative to prior clinical experience.

The company says it will meet with FDA later this year to finalize its regulatory pathway. BBP-418 holds orphan drug, fast track, and rare pediatric disease designations in the U.S., positioning it for potential priority review voucher eligibility if approved.