Story perspectives

FDA Rejects Disc Medicine's Bitopertin Over Approval Concerns

2/14/2026

Summary

Story summary

The U.S. Food and Drug Administration rejected Disc Medicine's bitopertin for erythropoietic protoporphyria due to surrogate-endpoint concerns.
The agency noted bitopertin met primary goals but the link between protoporphyrin reduction and clinical benefit remains unclear.
This decision underscores ongoing scrutiny of rare-disease approvals.
Disc Medicine will address concerns in the Phase 3 Apollo trial, with results expected in late 2026.
Disc Medicine remains optimistic about future approval.

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Story summary

The U.S. Food and Drug Administration rejected Disc Medicine's bitopertin for erythropoietic protoporphyria due to surrogate-endpoint concerns.
The agency noted bitopertin met primary goals but the link between protoporphyrin reduction and clinical benefit remains unclear.
This decision underscores ongoing scrutiny of rare-disease approvals.
Disc Medicine will address concerns in the Phase 3 Apollo trial, with results expected in late 2026.
Disc Medicine remains optimistic about future approval.