Rare Disease Drug Data Falsified, Journal Retracts Study

The New England Journal of Medicine has retracted a significant clinical trial article that underpinned the approval of Amgen's rare-disease drug, Tavneos. This decision, made on June 29 (as of 2026-06-30T00:42:00+00:00), stems from serious concerns that patient outcome data were manipulated and that some researchers involved in the study were unblinded.

The journal disclosed that two academic authors requested the retraction after a U.S. Food and Drug Administration investigation uncovered alterations in results for nine patients. Additionally, it was found that some researchers had been informed about which patients were receiving Tavneos and which were not, with these discrepancies not being disclosed in the original publication.

This development occurs as the FDA's Center for Drug Evaluation and Research proposed withdrawing Tavneos' approval in April (as of 2026-06-30T00:42:00+00:00) due to a perceived lack of proven effectiveness and inaccuracies in its initial application. These concerns were further amplified in March (as of 2026-06-30T00:42:00+00:00) when the agency identified 76 cases of drug-induced liver injury potentially linked to Tavneos, including seven severe cases of vanishing bile duct syndrome and eight reported deaths.

Amgen, which has previously expressed confidence in Tavneos' benefit-risk profile, is actively engaging in efforts to validate the drug's efficacy. The company has enlisted a research firm for an independent review of Tavneos data ahead of a critical hearing with the FDA. Meanwhile, Europe's drug regulator last week also recommended revoking the drug's marketing authorization, citing similar worries regarding the integrity of its trial data.