BBP-418 - (FORTIFY)

May 27, 2026: BridgeBio Pharma, Inc. (NASDAQ: BBIO) said the FDA accepted for filing its NDA for oral BBP-418 to treat limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) and granted Priority Review. The PDUFA target action date is November 27, 2026; the FDA is not currently planning to hold an advisory committee meeting. BridgeBio stated it is poised to launch BBP-418 upon approval and is engaging European regulators to identify an expedited path to approval.

The NDA is supported by the Phase 3 FORTIFY trial: at the pre-specified 12-month interim analysis, BBP-418 met all primary and secondary endpoints, with treated participants 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.

BBP-418 previously received FDA Orphan Drug, Fast Track, and Rare Pediatric Disease designations, and EMA Orphan Drug designation. If approved, BridgeBio said BBP-418 could be the first approved therapy for LGMD2I/R9 and potentially the first for any form of LGMD, and may qualify the company for a Priority Review Voucher. BridgeBio also plans near-future studies in LGMD2I/R9 patients under 12 and in LGMD2M/R13 and LGMD2U/R20.