Amgen has requested a hearing with the U.S. Food and Drug Administration to defend its rare disease drug Tavneos, as the agency pushes for the drug’s withdrawal from the market due to safety concerns, according to a July 24 statement by the company. The request follows an earlier submission of data and analyses by Amgen aimed at discussing Tavneos’ future with regulators.
“Amgen strongly disagrees with the FDA’s proposal to withdraw Tavneos from the U.S. market,” the company said in its statement issued Friday. The trouble began in January when the FDA asked Amgen to pull Tavneos over liver toxicity issues that affected its risk-benefit profile. In April, reports emerged that 20 patients in Japan had died after taking Tavneos, most related to vanishing bile duct syndrome, a complication of drug-induced liver injury. Following these events, both U.S. and European regulators recommended withdrawing Tavneos from their markets, and a key study supporting approval was withdrawn from The New England Journal of Medicine.
Tavneos was approved in 2021 for ANCA-associated vasculitis (AAV), a rare autoimmune kidney disease causing inflammation of blood vessels leading to organ damage. Patients typically receive corticosteroids for treatment but face significant side effects such as increased infection risk and cardiovascular complications.
Amgen acquired Tavneos through its $3.7 billion purchase of ChemoCentryx in 2022. Despite mounting regulatory scrutiny—Tavneos accounted for just one percent of Amgen’s 2025 revenue—the company continues defending it, arguing that it met regulatory requirements for effectiveness and maintains a favorable benefit-risk profile.
In support of its case, Amgen cited real-world data from more than 2,200 patients across 71 published studies showing benefit-risk support for Tavneos. An independent re-adjudication by Duke Clinical Research Institute confirmed similar remission rates between Tavneos and prednisone at week 26 but did not show superiority at week 52; however, patients on Tavneos reduced glucocorticoid use more than those on prednisone.
The company also reviewed post-marketing safety data indicating about 31 serious hepatic adverse events per 1,000 patient years—mostly reversible laboratory abnormalities—with some deaths primarily among Japanese patients over age 65 within three months of starting therapy. In response to ongoing safety signals, Amgen updated labeling in May this year with clearer warnings about liver toxicity.
