BridgeBio Pharma, Inc. (BBIO) announced on Tuesday that the FDA has granted Rare Pediatric Disease Designation for BBP-418 for the treatment of Limb-girdle Muscular Dystrophy Type 2I/R9 also known as LGMD2I/R9.
The company also mentioned that it has exceeded its interim analysis enrollment target and anticipates top-line interim data from its Phase 3 registrational study known as FORTIFY, in individuals with LGMD2I/R9 by 2025. The study involves a planned interim analysis at 12 months that focuses on evaluating glycosylated aDG as a surrogate endpoint to back Accelerated Approval.
If BBP-418 receives approval, BridgeBio could be eligible for a Priority Review Voucher based on obtaining the Rare Pediatric Disease Designation.
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June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.