Overview
- The New England Journal of Medicine retracted the 2021 ADVOCATE trial paper after an FDA probe found that outcomes for nine patients were altered and that some researchers were unblinded, a decision reported on June 29.
- The EMA's CHMP concluded the ADVOCATE study breached good clinical practice, recommended revoking Tavneos' EU marketing authorisation, and advised stopping new starts and switching current patients to alternatives while enhancing liver monitoring.
- The FDA's Center for Drug Evaluation and Research proposed withdrawing Tavneos in April after finding manipulated trial results and citing safety concerns, including 76 U.S. cases of drug-induced liver injury with seven cases of vanishing bile duct syndrome and eight deaths.
- Amgen has hired an independent research firm and the Duke Clinical Research Institute to re-examine the trial data and will submit findings ahead of regulatory hearings as it seeks to defend the drug's benefit-risk profile.
- Patients and clinicians face urgent choices: regulators recommend intensified liver testing and transitioning to other therapies, the European Commission will make a final revocation decision, and U.S. actions could lead to market removal and broader changes in how regulators vet trial conduct.