FDA’s Avacopan Alert Raises More Questions Than It Answers
WASHINGTON, D.C – The Food and Drug Administration (FDA) today issued a Drug Safety Communication about avacopan (TAVNEOS), an oral drug for a group of rare rheumatologic diseases that cause inflammation of small-to-medium sized blood vessels. Collectively, the diseases are known as severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis. The alert highlighted serious postmarketing cases, including patient deaths, from drug induced liver injury associated with avacopan, first approved in the U.S. in 2021. Although the risk of liver toxicity was identified in clinical trials before the drug was marketed and included in the prescribing information, the fatal liver injury cases are new safety concerns. Of 76 cases “with reasonable evidence of a causal association with avacopan use,” 74 reported a serious outcome, including 54 hospitalizations and eight deaths, the agency said.
In February, Amgen disclosed that “On January 16, 2026, the FDA requested that ChemoCentryx voluntarily withdraw TAVNEOS from the U.S. market.” Amgen acquired ChemoCentryx in October 2022.
Avacopan is used together with glucocorticoids and other standard of care medications to treat adults with ANCA-associated vasculitis; it does not eliminate glucocorticoid use. The FDA recommended close monitoring of patients on avacopan, including liver panel testing every two weeks in the first month of treatment and monthly for the next five months, and prompt discontinuation of the drug for symptoms or laboratory evidence of liver disease.
Dr. Robert Steinbrook, Health Research Group Director at Public Citizen, issued the following statement:
“The FDA’s Drug Safety Communication about avacopan highlighting the eight fatal cases of drug-induced liver injury and more than 50 hospitalizations is important but raises more questions than it answers.
“If the FDA in January 2026 requested that avacopan be voluntarily withdrawn from the U.S. market, as Amgen has disclosed, why has the agency not made this request publicly? Why is the FDA not publicly pushing back against the company for failing to withdraw the drug? Why does the prescribing information for avacopan not include a boxed warning for the risk of fatal liver disease? Does the FDA have data to establish that its recommendations for close monitoring of patients taking avacopan will prevent severe or fatal drug-induced liver injury? The FDA and Amgen must promptly answer these urgent questions.”