Disc Medicine Aligns with FDA on Path Forward for Bitopertin in EPP
Event summary
- Disc Medicine completed a Type A meeting with the FDA to discuss the CRL for bitopertin in EPP.
- The FDA agreed that a successful Phase 3 APOLLO study could support a traditional approval.
- Disc plans to submit a CRL response by the end of 2026, with an FDA decision expected by mid-2027.
- The APOLLO study is a double-blind, placebo-controlled Phase 3 trial with sites in the US, Canada, Europe, and Australia.
- Bitopertin is an investigational GlyT1 inhibitor licensed from Roche in May 2021.
The big picture
Disc Medicine's alignment with the FDA on the path forward for bitopertin in EPP marks a critical step in the regulatory process. The company's focus on hematologic diseases and its portfolio of innovative therapeutic candidates aim to address a wide spectrum of conditions by targeting fundamental biological pathways. The outcome of the APOLLO study and the subsequent FDA decision will be pivotal in determining the future of bitopertin as a potential treatment option for patients with EPP.
What we're watching
- Regulatory Clarity
- How the FDA's alignment on the APOLLO study's potential to support traditional approval will impact Disc's regulatory strategy.
- Clinical Execution
- Whether Disc can successfully complete the APOLLO study and report positive data by Q4 2026.
- Market Opportunity
- The pace at which bitopertin could become the first disease-modifying therapy for erythropoietic porphyrias if approved.