US FDA warns of liver injury cases tied to Amgen's rare disease drug

Core Viewpoint - The U.S. FDA has identified cases of liver injury linked to Amgen's drug Tavneos, prompting a warning for healthcare providers to monitor patients closely and discontinue treatment if liver damage is suspected [1][2]. Group 1: FDA Findings - The FDA reported 76 cases of drug-induced liver injury associated with Tavneos, including seven cases of vanishing bile duct syndrome, which can lead to permanent liver damage [2]. - Among the reported cases, there were eight fatalities [2]. Group 2: Regulatory Scrutiny - The safety warning adds to the regulatory scrutiny surrounding Tavneos, which is approved for treating ANCA-associated vasculitis, a rare autoimmune disease [3]. - In January, the FDA requested Amgen to voluntarily withdraw Tavneos due to concerns about the integrity of primary endpoint data from a late-stage trial involving 331 patients [3][4]. Group 3: Company Response - Amgen declined to withdraw Tavneos, asserting confidence in the drug's benefit-risk profile and indicating ongoing collaboration with the FDA [4]. - The median time to onset of drug-induced liver injury was reported as 46 days after starting treatment [4]. Group 4: International Regulatory Actions - While Tavneos labels in Europe and Australia mention post-marketing cases of vanishing bile duct syndrome, the U.S. prescribing information does not currently include this warning [5]. - The European Medicines Agency has initiated a review of Tavneos due to emerging concerns regarding data integrity [5].