FDA Targeting the Safety of Avacopan Save

The future of avacopan is up in the air. In April, the FDA issued a drug safety warning, previously requesting that Amgen to pull its rare disease drug Tavneos from the market. Now, there appears more pressure to remove it from the market.

The FDA’s concern is that the data, orginally generated by Chemocentryx,  was "manipulated" during the FDA submission.

CDER has sent a letter to Amgen’s citing its concerns, including that the drug may mot be effective and that its application for approval to treat ANCA-associated vasculitis included untrue statements. In particular, the FDA has become “increasingly concerned about the safety of Tavneos,” pointing to cases of serious drug-induced liver injury (DILI).

Just last month, the FDA warned of the risk of liver injury that could accompany use of Tavneos. Since its approval and through October 2024, 76 cases of DILI with “reasonable evidence” of a causal association to Tavneos were reported to the FDA Adverse Event Reporting System (FAERS). Nearly all of the reported cases resulted in a serious outcome, including hospitalization in 54 patients and death in eight, the FDA said.

Avacopan is used with glucocorticoids and other standard-of-care medications to treat the rare autoimmune disease anti-neutrophil cytoplasmic antibody-associated (ANCA) vasculitis, which causes damage to small blood vessels and eventually leads to organ failure, especially in the kidneys.