RARE Daily

FDA Grants Fast Track Designation to BridgeBio’s Experimental Therapy for LGMD2i

September 15, 2021

The U.S. Food and Drug Administration granted Fast Track designation to BridgeBio Pharma for the investigation of BBP-418 as a treatment option for limb-girdle muscular dystrophy type 2i.

Photo: Douglas Sproule, chief medical officer of ML Bio Solutions

Limb-girdle muscular dystrophy (LGMD2i) is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is a critical enzyme that adds a specific sugar molecule to a muscle cell structural protein called alpha-dystroglycan (αDG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack a robust cushioning system that is provided by fully glycosylated αDG proteins. Pediatric and adult patients with LGMD2i most commonly present with upper and lower extremity (“limb”) and thoracic (“girdle”) dysfunction (“limb-girdle” pattern of weakness), and without treatment often develop additional severe clinical manifestations, including loss of independent ambulation, severe breathing issues which can require mechanical ventilation, cardiomyopathy, and premature death.

“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, chief medical officer of ML Bio Solutions, 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.”

BBP-418 is being investigated as a treatment for LGMD2i. It is designed to overcome the enzymatic limitation of the defective FKRP enzyme by supplementing endogenous sugar molecules to glycosylate αDG and to improve muscle cell integrities, resulting in improved muscle strength and function for patients. Clinical trials to verify the safety and efficacy of BBP-418 are ongoing.

BBP-418 has received Orphan Drug designation for the treatment of LGMD2i from the FDA and for LGMD from the European Medicines Agency. BridgeBio is currently advancing its phase 2 clinical trial in subjects with a genetically confirmed diagnosis of LGMD2i. If the development program is successful, BBP-418 could be the first approved therapy for the treatment of patients with LGMD2i.

The FDA grants development programs Fast Track designation to help drive the development and expedite its review process for drugs being investigated to treat serious conditions and fill unmet medical needs. The FDA utilizes this program to provide patients access to important new drugs as early as possible. This is the fifth Fast Track designation for an investigational therapy that BridgeBio has received this year.

Author: Rare Daily Staff

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