THOUSAND OAKS, Calif. – Amgen has mounted a comprehensive defense of its rare disease drug Tavneos as it seeks to prevent the FDA from withdrawing the therapy from the U.S. market over safety concerns.
The company announced Friday that it had formally requested an FDA hearing after submitting a package of new analyses and evidence supporting Tavneos’ benefit-risk profile. In a statement, Amgen said it “strongly disagrees” with the agency’s proposal to withdraw the drug, arguing that Tavneos met regulatory standards for demonstrating efficacy and continues to provide meaningful benefits for patients with severe active ANCA-associated vasculitis (AAV).
“We welcome the opportunity for a science- and patient-focused engagement with the FDA, where we will present the totality of scientific evidence supporting Tavneos,” the company said. Amgen emphasized the seriousness of AAV, the limitations of existing treatment options, and the importance of preserving therapeutic choices for patients with this rare autoimmune disease.
FDA scrutiny intensifies
Tavneos, which received FDA approval in 2021 for AAV, has come under increasing regulatory pressure this year. In January, the FDA asked Amgen to voluntarily withdraw the drug, citing concerns that liver toxicity altered its overall benefit-risk profile.
Those concerns intensified in April after reports that 20 patients in Japan had died after receiving Tavneos, which is marketed there by Amgen’s partner, Kissei Pharmaceutical. Most of the deaths were associated with vanishing bile duct syndrome (VBS), a severe complication of drug-induced liver injury (DILI). Although Kissei briefly advised physicians to stop prescribing the drug to new patients, it later reversed that recommendation.
Since then, both the FDA and the European Medicines Agency have recommended that Tavneos be withdrawn from the market. Meanwhile, the pivotal ADVOCATE trial that supported the drug’s approval was retracted by The New England Journal of Medicine.
Amgen acquired Tavneos in 2022 through its $3.7 billion acquisition of ChemoCentryx.
New evidence in support of Tavneos
To support its case, Amgen submitted evidence from 71 published real-world studies involving more than 2,200 patients, arguing that the collective data continue to demonstrate a favorable benefit-risk profile.
The company also sponsored an independent re-analysis of the ADVOCATE trial through the Duke Clinical Research Institute. According to Amgen, the independent adjudication confirmed that Tavneos was non-inferior to prednisone in achieving remission at Week 26 and sustained remission at Week 52.
Patients receiving Tavneos also experienced substantially lower glucocorticoid exposure, with an average 81% reduction in steroid use compared with a 56% reduction among patients treated with prednisone.
Amgen acknowledged that the independent review did not demonstrate superiority over prednisone at Week 52. However, the company said the findings were broadly consistent with the original ADVOCATE results despite the scrutiny surrounding the study.
In addition, Amgen cited real-world evidence showing high rates of disease control, including a reported 93% remission rate after one year of Tavneos treatment.
Safety profile and label updates
Amgen also presented updated post-marketing safety data, reporting approximately 31 serious hepatic adverse events per 1,000 patient-years of exposure. While most cases involved reversible liver enzyme abnormalities, the company acknowledged that some patients—particularly Japanese patients older than 65 years—developed VBS, including fatal cases. According to Amgen, all reported VBS events occurred within the first 90 days of treatment.
The company noted that hepatotoxicity had already been identified during Tavneos’ clinical development program and that liver monitoring recommendations have been included in the U.S. prescribing information since the drug’s initial approval. Amgen proposed strengthened liver safety warnings in 2024, and the updated label became effective on May 29.
Patient need remains central
AAV is a rare autoimmune disease that causes inflammation of small blood vessels and can lead to severe organ damage, including kidney failure. Standard treatment relies heavily on corticosteroids, which are effective but carry substantial long-term risks, including serious infections, osteoporosis, diabetes, cardiovascular disease, and weight gain.
Amgen argued that Tavneos offers patients an opportunity to reduce prolonged steroid exposure while maintaining disease control. The company included patient testimonials describing the challenges of long-term steroid use and the benefits of tapering corticosteroids after starting Tavneos.
“Against this backdrop, patients and caregivers have described Tavneos as meaningful not only because of its role in disease management, but also because it may help reduce reliance on prolonged steroid exposure when used appropriately with standard therapy,” Amgen said.
Summarizing its position, the company argued that Tavneos should be evaluated using the totality of available evidence—including independent re-analysis of the ADVOCATE trial, real-world clinical experience, post-marketing safety data, patient needs, and physician judgment. Based on that body of evidence, Amgen maintains that Tavneos remains an important treatment option for appropriately selected patients with AAV despite ongoing regulatory concerns.
Media Contact
Investors Contacts
Phone: +1 805-447-1060
Email: [email protected]