Key Takeaways
- Disc Medicine has finalized the design for a confirmatory trial of its drug candidate bitopertin for erythropoietic protoporphyria (EPP).
- The FDA requested the addition of a co-primary endpoint to enhance the trial’s robustness in pursuit of accelerated approval.
- Enrollment for the 150-patient study is expected to progress significantly before the FDA’s decision on accelerated approval.
Trial Design Finalization for EPP Drug Candidate
Disc Medicine has announced that it has finalized the design of a confirmatory trial for its drug candidate, bitopertin, aimed at treating erythropoietic protoporphyria (EPP), a rare genetic disorder that leads to heightened sensitivity to sunlight. This trial design comes at the request of the U.S. Food and Drug Administration (FDA), which recommended the inclusion of a co-primary endpoint to streamline the pathway toward potential accelerated approval.
The company plans to submit an application for accelerated approval in the second half of this year. Last year, Disc entered discussions with the FDA regarding surrogate endpoints, ultimately agreeing on the reduction of protoporphyrin IX (PPIX) as a surrogate measure. This has become a key part of their trial.
Under the final design of the trial, the primary endpoint will still include the average monthly time patients can be exposed to sunlight without experiencing pain, which has been established in previous dialogues with the FDA. The newly introduced co-primary endpoint focuses specifically on changes in whole blood metal-free PPIX levels after a six-month treatment period.
Dr. Will Savage, chief medical officer of Disc Medicine, highlighted the significance of the co-primary endpoint during a recent conference call with investors. He expressed optimism about meeting this objective due to its highly objective nature, asserting that the FDA’s inclusion of PPIX as a co-primary endpoint validates its crucial role in the treatment’s evaluation. “The elevation of this endpoint to co-primary is an indication that regulators understand the importance of the role of PPIX,” Dr. Savage stated.
To gain accelerated approval, Disc Medicine will need to demonstrate achievement of both primary endpoints during the ongoing trial. The study aims to involve 150 patients, and the company anticipates that patient enrollment will be substantially progressed before the FDA’s evaluation of the accelerated approval application.
In summary, Disc Medicine is moving forward with an updated trial design for bitopertin in response to FDA feedback, reinforcing its commitment to meeting critical endpoints that could expedite the drug’s potential approval for treating EPP. The incorporation of both primary endpoints aims to strengthen the evidence supporting the efficacy of bitopertin for patients with this severe genetic condition.
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