FDA fast tracks limb-girdle muscular dystrophy type 2i treatment

BridgeBio Pharma Inc. announced it has received FDA fast track designation for BBP-418 as a treatment option for limb-girdle muscular dystrophy type 2i.

“As of now, there are no approved treatment options for people born with limb-girdle muscular dystrophy type 2i,” Douglas Sproule, MD, MSc, chief medical officer of ML Bio Solutions Inc., the BridgeBio company developing BBP-418, said in a press release. “People living with this disease can lose their ability to perform routine daily activities and ultimately may lose the ability to walk, need ventilatory support or face the risk of heart failure.”

The treatment was designed to overcome the enzymatic limitation of the defective fukutin-related protein enzyme and to improve muscle cell integrities, thus improving patients’ muscle strength and function. Ongoing clinical trials seek to verify the safety and efficacy of BBP-418, according to the release.

The FDA has granted the investigational therapy orphan drug designation for treating limb-girdle muscular dystrophy (LGMD) type 2i, and the European Medicines Agency granted the therapy the same designation for LGMD. BridgeBio is currently advancing phase 2 clinical trials among individuals with a genetically confirmed LGMD2i diagnosis. BBP-418 would be the first approved therapy for treating patients with LGMD2i should the development program prove successful.

“We are grateful the FDA has granted our program fast track designation based on the potential of our investigational therapy to treat this very serious condition,” Sproule said in the release. “We are hopeful the designation will allow us to address this unmet medical need by allowing us to potentially deliver our medicine to patients more quickly.”