Treatment candidate for limp-girdle muscular dystrophy receives Rare Pediatric Designation
The company also announced that it has surpassed its interim analysis enrollment target and expects topline phase 3 data from the FORTIFY study (NCT05775848) among individuals with LGMD21/R9 in 2025.
Treatment candidate for limp-girdle muscular dystrophy receives Rare Pediatric Designation | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.
BridgeBio Phamra's limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) candidate BBP-418 has received Rare Pediatric Disease Designation according to a press release from the company.
LGMD21/R9 is a monogenic autosomal recessive disease caused by partial loss of function mutations in the fukutin-related protein (FKRP) gene. Manifestations generally present as a skeletal myopathy affecting lower then upper limbs. Typically, this is later accompanied by respiratory muscle and cardiac muscle involvement. Disease manifestations typically occur in late childhood with progression to independent ambulation, assisted ventilation, and cardiomyopathy in adulthood (25%, 10%, and 30%, respectively) according to BridgeBio.
The company also announced that it has surpassed its interim analysis enrollment target and expects topline phase 3 data from the FORTIFY study (NCT05775848) among individuals with LGMD21/R9 in 2025. The ongoing randomized, double-blind, placebo-controlled study aims to evaluate the safety and efficacy of BBP-418.
"The study includes a planned interim analysis at 12 months focused on assessing glycosylated alpha-dystroglycan (αDG) as a surrogate endpoint to support Accelerated Approval. The primary endpoint to be evaluated at 36 months is the North Star Assessment (NSAD) for limb-girdle type muscular dystrophies and is designed to provide confirmatory clinical data," wrote BridgeBio in the press release. The company continues to enroll for the trial in the United States, United Kingdom, Europe, and Australia.
“Reduced glycosylated αDG 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, MD, MSc, chief medical officer, ML Bio Solutions, a BridgeBio affiliate, in a statement.
"We hope to continue to serve individuals through expeditious enrollment of the Phase 3 FORTIFY study and look forward to continuing to partner with the FDA to accelerate development of a potential new therapeutic option for people living with LGMD2I/R9," Sproule said.
Reference:
Bridgebio pharma surpasses interim analysis enrollment target and receives u.s. fda rare pediatric disease designation for bbp-418, a potential treatment for limb-girdle muscular dystrophy type 2i/r9 (lgmd2i/r9). BridgeBio Pharma. Press release. June 18, 2024. Accessed June 18, 2024.
