April 27 (Reuters) – The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) has proposed withdrawing approval of Amgen’s drug for rare autoimmune diseases, citing a lack of proven effectiveness and false statements in its original application.
In March, the agency identified 76 cases of drug-induced liver injury with evidence suggesting a causal link to Tavneos, including seven cases of vanishing bile duct syndrome (VBDS), a rare condition that can cause permanent liver damage. Eight deaths were reported among those cases.
A senior official at the CDER, which regulates prescription and over-the-counter medicines, said in a letter to Amgen on Monday that the agency can no longer conclude that “there is, or has ever been, a valid demonstration of substantial evidence of effectiveness for TAVNEOS.”
In the letter, CDER proposed to withdraw the drug “because of new information, which was withheld from FDA and did not become known to FDA until more than three years after approval, indicating that there is a lack of substantial evidence of effectiveness for the drug, and the application contains untrue statements of material facts.”
Amgen said they remained confident in TAVNEOS as a safe and effective medicine, adding that it was supported by real-world evidence and years of clinical data.
“Our perspective on the benefit-risk profile of TAVNEOS differs from the Agency’s. We will evaluate next steps and respond to the FDA,” a spokesperson for the company told Reuters in an emailed statement.
Tavneos, approved in October 2021, treats anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, a rare group of autoimmune diseases that cause inflammation in small- to medium-sized blood vessels.
The FDA had previously asked Amgen to voluntarily withdraw the drug from the U.S. market amid concerns about trial data, a request the company declined. At the time, Amgen said it was not aware of any problems with the underlying patient data, remained confident in Tavneos’ benefit-risk profile, and was working with the FDA on next steps.
Tavneos will remain on the market unless Amgen withdraws it or the FDA commissioner orders its removal, the agency said. In the meantime, the FDA advised healthcare professionals to discuss Tavneos and alternative treatment options with patients while a final decision on the drug’s marketing status is pending.
(Reporting by Natalia Bueno Rebolledo and Mrinmay Dey in Mexico City; Additional reporting by Chandni Shah in Bengaluru; Editing by Rashmi Aich)
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