Avacopan faces regulatory scrutiny over efficacy data and liver safety

Regulatory scrutiny is increasing for the autoimmune disease drug Avacopan (Tavneos) due to concerns regarding data integrity and patient safety.

In the United Kingdom, the Medicines and Healthcare products Regulatory Agency (MHRA) has suspended the use, supply, and sale of Avacopan to new patients as of September 1. This decision follows a reassessment which concluded that the clinical study supporting the drug’s efficacy for treating granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) is no longer considered reliable. While existing patients will undergo a six-month managed withdrawal period to transition to alternative treatments, the MHRA intends to revoke the medicine’s UK marketing authorisation by March 2027.

In the United States, a registry analysis of over 200 patients has identified safety concerns regarding liver injury. The study, published in Arthritis & Rheumatology, noted cases of drug-induced liver injury (DILI) and significant liver-enzyme elevations. This follows previous FDA reports of postmarketing cases of DILI, including fatalities and vanishing bile duct syndrome. Researchers emphasize the necessity of close liver-function monitoring for patients receiving the C5a receptor inhibitor.