Amgen announced Friday that it filed new evidence with the U.S. Food and Drug Administration on July 23, as part of its effort to secure a hearing and fight back against a proposed ban on its rare-disease medication Tavneos.
The FDA first proposed pulling Tavneos from American pharmacy shelves back in April. Regulators cited two main concerns: that the drug had not been proven effective enough, and that Amgen had made false statements in the application it originally submitted to get the drug approved.
Tavneos is used to treat severe active ANCA-associated vasculitis, a rare autoimmune disorder in which the body’s immune system attacks blood vessels, potentially causing serious damage to organs such as the kidneys and lungs.
Amgen said it firmly disagrees with the FDA’s position. The company’s new submission includes data from more than 70 real-world studies covering over 2,200 patients, all of which Amgen says support the drug’s effectiveness and safety record.
As part of its case, Amgen commissioned the Duke Clinical Research Institute to conduct an independent review of the drug’s key clinical trial. That review found that Tavneos performed comparably to a steroid-based treatment at both 26 and 52 weeks — though the independent review did not replicate the original trial’s conclusion that Tavneos was superior at the 52-week mark.
Safety concerns have also surfaced around the drug. In March, the FDA separately flagged 76 cases of serious liver injury connected to Tavneos, including eight patient deaths and seven cases of a rare condition capable of causing permanent liver damage.
The situation has drawn international attention as well. In June, Europe’s drug regulatory body recommended revoking Tavneos’ approval there, pointing to what it described as unreliable study data.
Despite all of this, the FDA noted in April that Tavneos will continue to be available in the United States unless Amgen voluntarily removes it or the FDA commissioner issues a formal order to pull it from the market. The FDA had not responded to a request for comment at the time of this report.
