Disc Medicine Announces Submission of New Drug Application (NDA) to US FDA for Accelerated Approval of Bitopertin for Patients with Erythropoietic Protoporphyria (EPP)

Core Viewpoint - Disc Medicine, Inc. has submitted a New Drug Application (NDA) to the FDA for bitopertin, targeting patients aged 12 and older with erythropoietic protoporphyria (EPP), aiming for accelerated approval due to significant unmet medical needs [1][2][3] Company Overview - Disc Medicine is a clinical-stage biopharmaceutical company focused on developing novel treatments for serious hematologic diseases, with a portfolio targeting heme biosynthesis and iron homeostasis [9] Product Details - Bitopertin is an investigational, orally administered inhibitor of glycine transporter 1 (GlyT1), designed to modulate heme biosynthesis and potentially serve as the first disease-modifying therapy for erythropoietic porphyrias [4][5] - The NDA submission is supported by Phase 2 BEACON and AURORA studies, which showed significant reductions in protoporphyrin IX (PPIX) and improvements in light tolerance and quality of life for EPP patients [2][4] Regulatory Pathway - The NDA includes a request for Priority Review, which could shorten the FDA review period to six months, highlighting the potential for significant improvement in treatment effectiveness [3][7] Disease Context - Erythropoietic protoporphyria (EPP) is a rare and debilitating disease caused by mutations affecting heme biosynthesis, leading to severe reactions to sunlight and potential liver complications [6][8]