FDA Fast Track designation for limb-girdle muscular dystrophy 

BridgeBio has been granted Fast Track designation United States Food and Drug Administration (FDA) for the investigation of BBP-418 as a treatment option for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i). This is the fifth Fast Track designation for an investigational therapy that BridgeBio has received in 2021. 

BridgeBio’s LGMD2i investigational therapy is one of the company’s 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and genetically-driven cancers. 

BridgeBio’s first wave of programmes are the now-approved drugs for Molybdenum Cofactor Deficiency (MoCD) Type A and previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement. The second wave of programmes includes the company’s four major near-term catalysts for its product candidates for the treatment of transthyretin (TTR) amyloidosis (ATTR), achondroplasia, congenital adrenal hyperplasia (CAH) and autosomal dominant hypocalcemia type 1 (ADH1). 

LGMD2i represents one of the leading programmes in BridgeBio’s ongoing third wave in development, which includes a variety of programmes in the cancer and mendelian space already in the clinic. 

With approximately 7,000 patients with potentially treatable mutations, 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. The investigational therapy 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 II 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. 

Image credit: Kate Hliznitsova

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