On Feb. 23, FDA announced its final decision to withdraw approval of Pepaxto (melphalan flufenamide), which was approved for use in combination with dexamethasone to treat certain patients with multiple myeloma.
The agency determined the following grounds for withdrawal were met: (1) the confirmatory study conducted as a condition of accelerated approval did not confirm Pepaxto’s clinical benefit, and (2) the available evidence demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use.
The final decision was issued by FDA’s Center for Biologics Evaluation and Research Director Peter Marks, M.D., Ph.D., as the Commissioner’s designee. The decision is effective immediately. FDA plans to publish a Federal Register notice announcing the availability of today’s decision and remove Pepaxto from the Orange Book. It is FDA’s understanding that Oncopeptides does not currently market Pepaxto in the U.S.
This is the first time FDA has used the amended procedures for withdrawal of accelerated approval that were enacted in 2023, as part of the Food and Drug Omnibus Report Act of 2022 (FDORA). In accordance with the new procedures, FDA provided Oncopeptides with a notice of proposed withdrawal of approval, an explanation for the proposed withdrawal, and an opportunity for a meeting and a written appeal to the Commissioner (or designee). Oncopeptides submitted a written appeal and met with the Commissioner’s designee. This decision is FDA’s response. See the public docket for more information.
FDA has the press release.
