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]

Core Viewpoint - Disc Medicine, Inc. received positive feedback from the FDA regarding its planned New Drug Application (NDA) submission for bitopertin, aimed at treating Erythropoietic Protoporphyria (EPP) [1][2] Group 1: NDA Submission Details - The pre-NDA meeting confirmed alignment with the FDA on the timing and content of the NDA submission, which is scheduled for October 2025 [2] - The NDA will be submitted under the FDA's accelerated approval pathway based on existing data [2] Group 2: About Bitopertin - Bitopertin is an investigational, orally administered inhibitor of glycine transporter 1 (GlyT1), designed to modulate heme biosynthesis [3] - It has potential as a first disease-modifying therapy for various hematologic diseases, including EPP [3] - Bitopertin has undergone multiple clinical trials, including the Phase 2 BEACON trial and the Phase 2 AURORA trial [3] Group 3: About Erythropoietic Protoporphyria (EPP) - EPP is a rare and potentially life-threatening disease caused by mutations affecting heme biosynthesis, leading to the accumulation of protoporphyrin IX (PPIX) [5] - Symptoms include severe pain and complications such as gallstones and liver damage, with current treatment options being limited [5] - There is currently only one FDA-approved therapy for EPP, Scenesse® (afamelanotide) [5] Group 4: About Disc Medicine - Disc Medicine is focused on discovering and developing novel treatments for serious hematologic diseases, targeting heme biosynthesis and iron homeostasis [6] - The company aims to build a portfolio of innovative therapeutic candidates to address a wide spectrum of hematologic diseases [6]