After the FDA flagged patient deaths linked to Amgen’s rare disease drug Tavneos and called for its voluntary removal, the pharma recruited an independent data analysis from Duke researchers to help build the case for the drug’s continued market approval.
Amgen has requested a hearing with the FDA over the agency’s insistence that Amgen pull its rare inflammatory disease drug Tavneos from the market—and this time, the company is bringing in help from the Duke Clinical Research Institute.
The pharma has asked Duke researchers to conduct an “independent and fully blinded re-adjudication” of data from the ADVOCATE trial, according to a June 1 letter to the FDA, which was posted online on Thursday. ADVOCATE is the Phase 3 study that Tavneos’ original developer ChemoCentryx used to support its approval in ANCA-associated vasculitis in October 2021. Amgen acquired ChemoCentryx for $4 billion just under a year later.
“Withdrawal of approval of TAVNEOS would be neither in the best interest of patients nor consistent with the statutory criteria,” Jay Bradner, Amgen’s executive vice president for R&D, wrote in the company’s letter to the FDA. Duke’s re-adjudication of ADVOCATE data began in February 2026 and the pharma plans to submit results, alongside other “detailed data,” to the FDA by June 29.
Alongside the Duke analysis, Amgen has listed in its letter more than a dozen ongoing or recently completed studies to support Tavneos’ overall benefit-risk profile.
The FDA first called for the voluntary withdrawal of Tavneos in January, raising concerns about “the process followed by ChemoCentryx” to evaluate the primary endpoints in ADVOCATE for nine of the 331 patients enrolled, according to an Amgen release in February. The regulator also pointed to liver toxicities in the context of Tavneos’ overall risk-benefit profile.
Amgen refused the regulator’s request, leaving Tavneos on the market.
The agency continued to apply pressure, however, and in late March put out a safety alert flagging eight deaths and 76 cases of drug-induced liver injury (DILI) that have “reasonable evidence of a causal association with Tavneos.” Seven of the 76 cases involved biopsy-confirmed vanishing bile duct syndrome (VBS), all of which necessitated hospitalization and three of which contributed to the total tally of eight deaths.
While the FDA at the time conceded that liver toxicities have been “identified in premarket clinical trials and described in product labelling,” potentially fatal DILI and VBS “represent new safety concerns.”
Last month, Japan’s Kissei Pharmaceutical, which is Amgen’s partner in charge of Tavneos’ Japanese distribution, said that 20 patients on the drug have died since the product launched in 2022. Most of the deaths were attributed to DILI and VBS, though Kissei took care to say that it remains unclear if Tavneos was the direct cause of the fatalities.
The European Medicines Agency has also launched a review of Tavneos, similarly flagging “questions regarding the data integrity” of ADVOCATE, according to a January release.
