Disc Medicine Announces Successful Type C Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria (EPP) and Shares Plans for NDA Submission

Core Viewpoint - Disc Medicine, Inc. received positive feedback from the FDA regarding the APOLLO post-marketing confirmatory trial for bitopertin in Erythropoietic Protoporphyria (EPP), marking progress towards potential accelerated approval and eventual full approval of the therapy [1][2][5] Group 1: FDA Interaction and Trial Plans - The FDA interaction is seen as a significant step towards providing a life-altering therapy for EPP patients, with plans to submit a New Drug Application (NDA) in the second half of 2025 [2][5] - The APOLLO trial is set to begin by mid-2025, with sites planned in the US, Canada, Europe, and Australia, and is expected to be well underway by the time of potential accelerated approval [3][5] - The Type C meeting clarified the design of the APOLLO trial, aligning on co-primary endpoints including average monthly time in light without pain and percent change in whole-blood metal-free protoporphyrin IX (PPIX) after six months of treatment [5][6] Group 2: Bitopertin Overview - Bitopertin is an investigational, orally administered inhibitor of glycine transporter 1 (GlyT1), aimed at modulating heme biosynthesis and potentially serving as the first disease-modifying therapy for hematologic diseases [4][8] - The drug has undergone multiple clinical trials, including the Phase 2 BEACON and AURORA trials, focusing on its efficacy in treating EPP [4][5] Group 3: Erythropoietic Protoporphyria (EPP) Context - EPP is a rare and debilitating disease caused by mutations affecting heme biosynthesis, leading to severe reactions to sunlight and significant impacts on patients' quality of life [8][9] - Current treatment options are limited, with only one FDA-approved therapy available, highlighting the need for new therapeutic options like bitopertin [8][9]