Amgen submits real-world patient data in bid to save Tavneos from FDA withdrawal

Amgen (AMGN) said it had submitted new evidence to the Food and Drug Administration as it seeks a formal hearing to challenge the proposed withdrawal of Tavneos, its treatment for a rare autoimmune condition that damages blood vessels, from the U.S. market.

The FDA proposed withdrawing Tavneos in April, citing a lack of proven effectiveness and false statements in the drug's original marketing application. The regulator had also separately identified 76 cases of serious liver injury linked to the drug, including eight deaths and seven cases of a rare condition that can cause permanent liver damage. Europe's medicines regulator followed in June, recommending the revocation of Tavneos's marketing authorization on the grounds of unreliable study data.

Amgen said it strongly disagrees with the FDA's position. Its July 23 submission includes more than 70 real-world studies involving over 2,200 patients that the company says support the drug's effectiveness and safety. The filing also incorporates findings from the Duke Clinical Research Institute, which Amgen had commissioned to conduct an independent review of the pivotal trial. Duke found that Tavneos matched a steroid treatment regimen at both 26 and 52 weeks, though the review did not reproduce the original trial's finding of superiority at 52 weeks.

Tavneos is approved to treat severe active ANCA-associated vasculitis, a rare disease that inflames blood vessels and can damage organs including the kidneys and lungs. An FDA official told Reuters that Amgen has until July 29 to submit all data, information and analyses in support of a hearing. The agency said in April that the drug would remain on the U.S. market unless Amgen voluntarily removes it or the FDA commissioner orders its withdrawal.