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Disc Medicine Announces Successful Type C Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria (EPP) and Shares Plans for NDA Submission
Pursuing accelerated approval for bitopertin in EPP with protoporphyrin IX (PPIX) reduction as the surrogate endpointPlanning to submit NDA under accelerated
About this update from Disc Medicine, Inc.
[{"type":"text","content":"Pursuing accelerated approval for bitopertin in EPP with protoporphyrin IX (PPIX) reduction as the surrogate endpointPlanning to submit NDA under accelerated approval pathway in H2 2025 based on existing clinical data, including results from BEACON and AURORA Phase 2 trialsAchieved regulatory alignment on APOLLO post-marketing confirmatory trial design and on track to initiate trial by mid-year 2025Aligned on average monthly time in light without pain during the last month of the 6-month treatment period and percent change from baseline in whole-blood metal-free PPIX after 6 months of treatment as coprimary endpoints for confirmatory trialManagement will host a conference call on Tuesday, January 21 at 8:00 am EST. WATERTOWN, Mass., Jan. 21, 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 positive feedback from its Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss the APOLLO post-marketing confirmatory trial for bitopertin in EPP. “Our recent FDA interaction marks another step toward delivering a potentially life-altering therapy for EPP patients, and we appreciate the collaboration with regulators, our investigators, and the EPP patient community which has brought us to this point,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “Last year, we aligned with the FDA on PPIX reduction as a surrogate endpoint for potential accelerated approval of bitopertin, and we are actively pursuing that path with plans to submit an NDA in the second half of 2025. As part of that process, the Type C meeting has provided further clarity on our plans for the APOLLO post-marketing confirmatory trial, which will kick off by the middle of this year and could eventually be the basis for converting an accelerated approval, if granted, to a full approval.” The meeting resulted in alignment on the design of the APOLLO post-marketing confirmatory trial. Key features include: Co-primary endpoints of average monthly total time in sunlight without pain between 10:00 and 18:00 during the last month of the 6-month treatment period and percent change from baseline in whole blood metal-free PPIX afte...