Disc Medicine receives CNPV from FDA for Bitopertin

Disc Medicine (IRON) announced that it received a Commissioner’s National Priority Voucher from the U.S. Food and Drug Administration for bitopertin in erythropoietic protoporphyria, including X-linked protoporphyria. Disc submitted a New Drug Application 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.”