Disc Medicine Earns FDA’s Commissioner’s National Priority Voucher For Bitopertin In EPP And XLP

Disc Medicine, Inc. , a clinical-stage biopharmaceutical company developing novel therapies for serious hematologic disorders, announced that it has received a Commissioner’s National Priority Voucher (CNPV) from the U.S. Food and Drug Administration (FDA) for bitopertin, a potential treatment for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). The company submitted a New Drug Application (NDA) for bitopertin in September 2025 under the FDA’s accelerated approval pathway for patients aged 12 years and older. Bitopertin has already earned Orphan Drug and Rare Pediatric Disease designations.

“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 CNPV program, introduced by the FDA in June 2025, aims to expedite the review of drugs that address critical U.S. health priorities. Under this initiative, qualifying therapies benefit from enhanced communication channels, rolling review, and a significantly shortened review period of 1–2 months. Receiving this voucher highlights the potential importance of bitopertin in addressing unmet medical needs in rare, debilitating conditions such as EPP.

Erythropoietic protoporphyria (EPP) is a rare and painful genetic disorder that disrupts heme biosynthesis, leading to a buildup of protoporphyrin IX (PPIX), a toxic compound that causes severe phototoxic reactions when exposed to sunlight. Patients experience intense pain, burning, swelling, and sometimes blistering, forcing many to avoid sunlight entirely. In severe cases, PPIX accumulation can damage the liver, leading to gallstones, cholestasis, or even liver failure. Current treatment options are largely preventive, relying on strict light avoidance and pain management, highlighting the urgent need for new therapeutic options like bitopertin.