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, 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).