Disc Medicine Announces Submission of New Drug Application (NDA) to US FDA for Accelerated Approval of Bitopertin for Patients with Erythropoietic Protoporphyria (EPP)

• Disc is seeking accelerated approval and priority review of its NDA submission
• FDA decision to accept and file the NDA for review occurs within 60 days of submission
  
  

WATERTOWN, Mass., Sept. 30, 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 the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bitopertin for patients aged 12 years and older with erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). Disc submitted the NDA under the FDA’s accelerated approval pathway using reduction of protoporphyrin IX (PPIX) as a surrogate endpoint and requested a Priority Review based on bitopertin’s potential to address the significant unmet need for EPP patients. Bitopertin has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.

“This NDA submission represents a pivotal moment not just for Disc but for the EPP community as we seek to provide patients with a treatment option that has the potential to address the underlying cause of disease,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “We look forward to working closely with regulators throughout the review process and remain focused on preparations for bitopertin’s anticipated launch. I want to express our gratitude to our dedicated investigators and the patients who participated in our clinical trials, and their families and caregivers who helped make this all possible.”

The NDA submission is supported by the results of the Phase 2 BEACON and AURORA studies in EPP, as well as prior data generated by Roche, including a safety database of over 4,000 clinical trial participants. The BEACON and AURORA studies demonstrated significant reductions in PPIX and improvements across key aspects of the disease, including improvements in light tolerance, reduction of phototoxic reactions, and improvements in quality of life. Disc is also studying bitopertin in a long-term extension study, HELIOS, and initiated the APOLLO confirmatory study in April 2025.