Disc Medicine, Inc. received a Complete Response Letter from the FDA for its New Drug Application for bitopertin as a treatment for erythropoietic protoporphyria, delaying potential approval. The FDA indicated the ongoing Phase 3 APOLLO study results could support traditional approval, with topline data anticipated in Q4 2026.
Key takeaways
FDA issued a Complete Response Letter for the NDA for bitopertin as a treatment for EPP on February 13, 2026.
FDA acknowledged AURORA and BEACON trials provided sufficient evidence that bitopertin significantly lowers PPIX.
FDA concluded trials did not show evidence of association between percent change in PPIX and sunlight exposure-based endpoints.
FDA indicated results of the ongoing Phase 3 APOLLO study could serve as evidence to support traditional approval.
Topline data from the APOLLO study is anticipated in Q4 2026.
Disc plans to request a Type A meeting with the FDA to review its approach.