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BBIO : FDA Grants Fast Track Designation For Therapy To Treat Limb-girdle Muscular Dystrophy Type 2i

BridgeBio Pharma Inc. (BBIO) said Wednesday that the U.S. Food and Drug Administration granted Fast Track designation for the investigation of BBP-418 as a treatment option for Limb-girdle Muscular Dystrophy Type 2i or LGMD2i.

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.

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 (aDG).

Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack a robust cushioning system that is provided by fully glycosylated aDG proteins.

Pediatric and adult patients with LGMD2i most commonly present with upper and lower extremity ("limb") and thoracic ("girdle") dysfunction , 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.

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