FDA rejects Disc Medicine's bitopertin over surrogate-endpoint concerns
TL;DR Summary
The FDA declined Disc Medicine’s bitopertin for erythropoietic protoporphyria, citing that the phase 2 trials used a surrogate endpoint (a ~40% drop in PPIX) whose link to meaningful clinical benefit—sun tolerance—remains unproven. The company plans to press on with the APOLLO phase 3 trial, which uses sun-exposure outcomes, while the broader regulatory environment and the CNPV priority voucher program continue to draw scrutiny.
Topics:business#bitopertin#disc-medicine#erythropoietic-protoporphyria#fda-rejection#health-biotechnology#surrogate-endpoint
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