BridgeBio announced a positive interim Phase 3 readout for BBP‑418 (ribitol) in limb‑girdle muscular dystrophy type 2I/R9, meeting prespecified thresholds and producing clinically meaningful improvements on a muscle‑stability marker. The company plans to meet with the U.S. Food and Drug Administration to discuss an NDA strategy, potentially seeking accelerated pathways. BridgeBio characterized the result as a 'home run' and said the data strengthen the regulatory case compared with prior expectations. Investors reacted favorably as the program could become the first approved therapy for this LGMD subtype. The outcome underscores the value of targeted rare‑disease trials where biomarker and functional endpoints can support expedited approval strategies.
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