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FDA rejects Disc Medicine's bitopertin over surrogate-endpoint concerns

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Source: Fierce Biotech
FDA rejects Disc Medicine's bitopertin over surrogate-endpoint concerns
Photo: Fierce Biotech
TL;DR

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:businesshealth-biotechnology#bitopertin#disc-medicine#erythropoietic-protoporphyria#fda-rejection#health-biotechnology#surrogate-endpoint
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  2. FDA rejects rare disease therapy from Disc Medicine, early recipient of commissioner’s voucher  statnews.com
  3. Drug For Rare Genetic Disorder Goes From FDA Priority to Reject in Four Months  Bloomberg.com
  4. FDA slaps CRL on Disc Medicine’s bitopertin NDA  The Pharma Letter
  5. Disc Medicine (IRON) Shares Plunge by 39% to $43.99  GuruFocus

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