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BridgeBio Pharma Receives FDA Fast Track Designation for Investigational Therapy for the Treatment of Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)
- If successful, BridgeBio's drug could be the first approved therapy for patients with LGMD2i - BridgeBio's investigational therapy for LGMD2i is one of more
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- If successful, BridgeBio's drug could be the first approved therapy for patients with LGMD2i\n - BridgeBio's investigational therapy for LGMD2i is one of more than 30 R&D programs in the company's diverse pipeline targeting genetic diseases and genetically-driven cancers\n - 12 of BridgeBio's programs are in the clinic and two have received FDA approval\n\n\nPALO ALTO, Calif., Sept. 15, 2021 /PRNewswire/ -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today 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.\n\n \n \n \n \n \n \n\n \nBridgeBio'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. \nBridgeBio'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).\nLGMD2i 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.\nWith approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (...