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Disc Medicine Receives FDA Orphan Drug Designation for Bitopertin for the Treatment of Erythropoietic Protoporphyria
WATERTOWN, Mass., Dec. 27, 2022 /PRNewswire/ -- Disc Medicine, Inc. ("Disc"), a clinical-stage biopharmaceutical company focused on the discovery,
About this update from Disc Medicine, Inc.
[{"type":"text","content":"WATERTOWN, Mass., Dec. 27, 2022 /PRNewswire/ -- Disc Medicine, Inc. (\"Disc\"), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced today that the U.S. Food and Drug Administration (\"FDA\") granted Orphan Drug Designation to bitopertin for the treatment of erythropoietic protoporphyria (\"EPP\"). Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 (\"GlyT1\") designed to modulate heme biosynthesis, and has been shown in preclinical studies to reduce accumulation of protoporphyrin IX (\"PPIX\"), the toxic metabolite that causes disease pathology in EPP patients. It is currently being studied in two ongoing Phase 2 studies in EPP, AURORA (NCT05308472) and BEACON (ACTRN12622000799752).\n\n \n \n \n \n \n \n\n \n\"Receiving orphan drug designation for bitopertin is incredibly encouraging and validates our commitment to bring a potential new treatment to EPP patients,\" said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. \"We are eagerly awaiting the results of our ongoing Phase 2 trials and look forward to collaborating with the FDA to progress bitopertin through clinical development.\"\nFDA Orphan Drug Designation may be granted to investigational drugs or biological products which show promise in treating rare medical diseases or conditions that affect fewer than 200,000 people in the United States. By receiving Orphan Drug Designation, bitopertin can benefit from certain development incentives and seven years of market exclusivity, subject to regulatory approval.\nAbout EPP\nEPP is a rare, debilitating and potentially life-threatening diseases caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate, PPIX. This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities ...