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BBP-418 Demonstrates Consistent Efficacy and Favorable Safety Profile in Phase 3 FORTIFY Interim Analysis in LGMD2I/R9
- Phase 3 FORTIFY interim analysis results presented in a late-breaking oral presentation at MDA highlight the broad and consistent efficacy of BBP-418 across
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- Phase 3 FORTIFY interim analysis results presented in a late-breaking oral presentation at MDA highlight the broad and consistent efficacy of BBP-418 across key clinical endpoints and prespecified subgroups - Early separation from placebo observed in 100MTT, with improvements in ambulation evident as early as three months following initiation of BBP-418 treatment - Based on the positive interim analysis results, BridgeBio intends to submit an NDA to the FDA for traditional approval in the first half of 2026 with a U.S. launch anticipated in late 2026/early 2027 - If successful, BBP-418 could be the first approved therapy for individuals living with LGMD2I/R9, potentially representing the first approval of a therapy for any form of LGMD PALO ALTO, Calif., March 11, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a biopharmaceutical company focused on developing medicines for genetic conditions, today presented additional positive data from the interim analysis of FORTIFY, the Phase 3 clinical trial of oral BBP-418, in individuals with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). These data were presented at the MDA Clinical and Scientific Conference in a late-breaking oral presentation by Katherine Mathews, M.D., Professor of Pediatrics and Neurology at the University of Iowa’s Roy J. and Lucille A. Carver College of Medicine. “People living with LGMD2I/R9 face a real unmet medical need. There is currently no specific treatment, the disease often has an early age of onset, and it is relentlessly progressive, leading to irreversible loss of motor function,” said Dr. Mathews. “The early separation from placebo on 100MTT is especially encouraging. In a progressive muscle disease where time matters, seeing early improvements in ambulation suggests a rapid onset of action of BBP-418. The improved motor and pulmonary functions seen in this interim analysis, together with a favorable safety profile, are exciting and suggest this will be a treatment with meaningful clinical impact.” New efficacy findings presented from the interim analysis include the following: Demographics, baseline characteristics, and baseline values across key endpoints were generally well balanced between the BBP-418 and placebo armsTreatment with BBP-418 resulted in a rapid and sustained reduction in serum...