BridgeBio Pharma, Inc. announced that the United States Food and Drug Administration (FDA) granted Fast Track designation for the investigation of BBP-418 as a treatment option for Limb-girdle Muscular Dystrophy Type 2i (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. 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 programs 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 programs 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 programs in BridgeBio's ongoing third wave in development, which includes a variety of programs 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 (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 ("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.