FDA Gives Fast Track Designation for Limb-Girdle Muscular Dystrophy Type 2i Therapy

The Food and Drug Administration (FDA) granted Fast Track designation for BBP-418 (BridgeBio Pharma, Palo, CA) as a treatment option for limb-girdle muscular dystrophy type 2i (LGMD2i). 

BBP-418 is a precursor of the sugar -dystroglycan (αDG) being investigated as a treatment for LGMD2i. Ribitol is designed to overcome the enzymatic limitation of the defective fukutin-related protein gene (FKRP) enzyme by supplementing endogenous sugar molecules and to improve muscle cell integrity and muscle strength and function. BBP-418 is currently advancing to phase 2 clinical trial in individuals with a genetically confirmed diagnosis of LGMD2i. With the success of the development program, BBP-418 could be the first approved therapy for the treatment of individuals with LGMD2i.

"As of now, there are no approved treatment options for people born with limb-girdle muscular dystrophy type 2i. 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," said Douglas Sproule, MD, MSc, chief medical officer of ML Bio Solutions, Inc., the BridgeBio company developing BBP-418. "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. 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."