Disc Medicine submits NDA to FDA for bitopertin in patients with EPP

Disc Medicine (IRON) announced the submission of a New Drug Application to the U.S. Food and Drug Administration for bitopertin for patients aged 12 years and older with erythropoietic protoporphyria, including X-linked protoporphyria. Disc submitted the NDA under the FDA’s accelerated approval pathway using reduction of protoporphyrin IX 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. 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 (RHHBY), 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. The NDA includes a request for Priority Review, which, if granted, would accelerate the timing of the FDA’s goal for review of the application to six months following the end of the 60-day filing review period rather than the standard 10-month review period. Priority Review status is designated for drugs that may offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious condition.