BridgeBio gets FDA rare pediatric disease designation for BBP-418 for MD

BridgeBio Pharma announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study FORTIFY in individuals with LGMD2I/R9 in 2025 “Reduced glycosylated aDG is the primary molecular driver of LGMD2I/R9, a serious, progressively debilitating disease that weakens the muscles causing many affected people to become fully dependent on a caregiver, while also threatening their cardiac and respiratory function,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Solutions…The company also announced that the FDA has granted Rare Pediatric Disease Designation RPDD for BBP-418 in the treatment of LGMD2I/R9, recognizing the rarity of this disease is characterized by serious manifestations primarily affecting children. If BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher based on receipt of the RPDD. A Priority Review Voucher can be applied to another therapy in the Company’s pipeline for a shorter timeline during the review process of a New Drug Application or can be sold and transferred to another company looking to receive priority