Disc Medicine receives FDA ODD for bitopertin

Disc Medicine announced that the U.S. Food and Drug Administration granted Orphan Drug Designation, ODD, to bitopertin for the treatment of erythropoietic protoporphyria. Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 designed to modulate heme biosynthesis, and has been shown in preclinical studies to reduce accumulation of protoporphyrin IX, the toxic metabolite that causes disease pathology in EPP patients. It is currently being studied in two ongoing Phase 2 studies in EPP, AURORA and BEACON. "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." In August 2022, Disc announced it entered into a definitive merger agreement with Gemini Therapeutics.