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WATERTOWN, Mass., Oct. 16, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced that it received a Commissioner's National Priority Voucher (CNPV) from the U.S. Food and Drug Administration (FDA) for bitopertin in erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). Disc submitted a New Drug Application (NDA) to the FDA for bitopertin for patients aged 12 years and older with EPP following the FDA's accelerated approval pathway in September 2025. Bitopertin has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.
"We are honored to have received this voucher which underscores the potential impact of bitopertin and reinforces the immense need EPP patients have for a new therapy that may address the underlying cause of their disease," said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. "We are grateful to the FDA for implementing this innovative new voucher program and we look forward to working closely with the Agency to bring bitopertin to patients as soon as possible. All of this would not be possible without collaboration from the EPP patient and caregiver community, and we thank them for their contributions."
The Commissioner's National Priority Voucher program, announced in June 2025, is designed to accelerate the development and review of certain drugs aligned with US national health priorities by offering the opportunity to reduce drug application review times to 1-2 months. Additionally, companies selected for the program will be issued a voucher entitling them to benefits including enhanced communications and rolling review to allow for shortened review time.
Erythropoietic protoporphyria is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families.
Posted In: IRON
