Disc Medicine Announces Receipt of FDA Commissioner’s National Priority Voucher (CNPV) for Bitopertin in Erythropoietic Protoporphyria (EPP)

Core Insights - Disc Medicine, Inc. has received a Commissioner's National Priority Voucher (CNPV) from the FDA for bitopertin, aimed at treating erythropoietic protoporphyria (EPP) [1][2] - The company submitted a New Drug Application (NDA) for bitopertin for patients aged 12 years and older with EPP, following the FDA's accelerated approval pathway [1][7] - Bitopertin has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, highlighting its potential significance for patients [1] Company Overview - Disc Medicine is a clinical-stage biopharmaceutical company focused on developing novel treatments for serious hematologic diseases [6] - The company aims to build a portfolio of innovative therapeutic candidates targeting heme biosynthesis and iron homeostasis [6] Product Details - Bitopertin is an investigational, orally administered inhibitor of glycine transporter 1 (GlyT1), designed to modulate heme biosynthesis [4] - It is being developed as a potential first disease-modifying therapy for erythropoietic porphyrias and has undergone multiple clinical trials, including the Phase 2 BEACON trial and the Phase 3 APOLLO trial [4][5] Disease Context - Erythropoietic protoporphyria is a rare and debilitating disease caused by mutations affecting heme biosynthesis, leading to severe reactions to sunlight [3] - Current treatment involves extreme measures to avoid sunlight exposure, significantly impacting patients' quality of life [3] Regulatory Insights - The CNPV program aims to accelerate drug application review times to 1-2 months, enhancing communication and allowing for rolling reviews [2][7] - The FDA's implementation of this program reflects a commitment to expedite the development of therapies aligned with national health priorities [2]