PALO ALTO, Calif. — October 5, 2026

BridgeBio Pharma, Inc. announced new exploratory cardiac findings from the 12-month interim analysis of the Phase 3 FORTIFY trial evaluating oral BBP-418 in individuals with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), an FKRP-related genetic disease. The data, presented at the 31st annual Congress of the World Muscle Society in Hiroshima, showed improvements in cardiac biomarkers and left ventricular ejection fraction (LVEF). Among participants with elevated high-sensitivity troponin I at baseline, 100% of BBP-418-treated participants returned to the normal range by Month 12 compared with 40% receiving placebo. BBP-418 is under FDA Priority Review with a PDUFA target action date of November 27, 2026.

BBP-418 Shows Potential Cardiac and Disease Biomarker Benefits

The FORTIFY analysis demonstrated improvements across several measures associated with cardiac and muscle disease activity. High-sensitivity troponin I declined more with BBP-418 than placebo, with a least-squares mean difference of -17.8 ng/L and a statistically significant p-value of 0.0443. At Month 12, 54% of participants receiving BBP-418 had stable or improved LVEF compared with 25% of placebo-treated participants. Separate analyses of glycosylated alpha-dystroglycan (αDG) also showed that BBP-418 increased mean glycosylated αDG to levels observed in asymptomatic heterozygous FKRP carriers by Month 3, with the effect sustained through Month 12. These findings support the potential for BBP-418 to address disease biology rather than solely manage symptoms.

FORTIFY Supports Potential Disease Modification

BBP-418 is designed to target the underlying mechanism of LGMD2I/R9 by enhancing residual FKRP enzyme activity and restoring αDG glycosylation. The oral glycosylation substrate therapy is intended to saturate the partially functional FKRP enzyme with additional substrate, supporting restoration of αDG glycosylation and potentially improving muscle function. In the Phase 3 FORTIFY trial, BridgeBio reported that BBP-418 met all primary and secondary endpoints at the prespecified 12-month interim analysis, with treated participants improving while placebo recipients declined across key measures. The company said the pattern of biomarker normalization, together with functional improvements, is consistent with the potential for BBP-418 to act as a disease-modifying therapy for LGMD2I/R9.

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BridgeBio Prepares for Potential BBP-418 Approval

BridgeBio is advancing BBP-418 toward a potential U.S. approval as it continues discussions with regulators in Europe. The company believes BBP-418 could become the first approved therapy for LGMD2I/R9 and potentially the first approved treatment for any form of limb-girdle muscular dystrophy. BBP-418 has received FDA Orphan Drug, Fast Track, Rare Pediatric Disease and Priority Review designations, as well as Orphan Drug designation from the European Medicines Agency. BridgeBio plans to initiate clinical studies in individuals younger than 12 years with LGMD2I/R9 in the first half of 2027 and intends to evaluate the therapy in additional FKRP-related conditions, including LGMD2M/R13 and LGMD2U/R20. With the FDA decision expected in November 2026, the latest FORTIFY data strengthen the clinical and biological rationale for BBP-418 as BridgeBio advances the program toward a potential regulatory milestone.

Source: BridgeBio Pharma press release