Disc Medicine Submits NDA To U.S. FDA Seeking Accelerated Approval Of Bitopertin For Patients With Erythropoietic Protoporphyria (EPP)

Disc Medicine, Inc., a clinical-stage biopharmaceutical company developing novel therapies for serious hematologic diseases, announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bitopertin. The application seeks approval for the treatment of patients aged 12 years and older with erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP).

The NDA was submitted under the FDA’s accelerated approval pathway, using reduction of protoporphyrin IX (PPIX) as a surrogate endpoint, and includes a request for Priority Review. Disc noted that bitopertin has the potential to address the significant unmet medical need in EPP, a rare and debilitating condition with limited treatment options. The FDA has previously granted Orphan Drug Designation and Rare Pediatric Disease Designation to bitopertin.

The submission is supported by results from the Phase 2 BEACON and AURORA studies in patients with EPP, which demonstrated significant reductions in PPIX levels and improvements in several clinical measures, including light tolerance, reduction of phototoxic reactions, and quality of life. Additional supporting data include Roche’s earlier studies of bitopertin, which generated a safety database involving more than 4,000 participants. Disc is continuing to study bitopertin in the long-term extension HELIOS trial and initiated the APOLLO confirmatory study in April 2025.

In a statement, John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc Medicine, described the NDA submission as a pivotal step for both the company and the EPP community. He emphasized the company’s commitment to working closely with regulators during the review process and highlighted preparations for the anticipated launch of bitopertin. He also expressed appreciation for the investigators, patients, and families involved in the clinical studies.

If granted, Priority Review would shorten the FDA’s review goal from the standard 10 months to 6 months following the conclusion of the 60-day filing review period. Priority Review is reserved for therapies that may significantly improve treatment, diagnosis, or prevention of serious conditions.