FDA withdraws approval of melphalan flufenamide

Excerpt

The US FDA has announced its final decision to withdraw approval of melphalan flufenamide [Pepaxto], which had received accelerated approval for use in combination with dexamethasone for the treatment of certain patients with multiple myeloma. FDA determined that the following grounds for withdrawal were met: the confirmatory study conducted as a condition of accelerated approval did not confirm the clinical benefit of Pepaxto; and available evidence demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use.The decision became effective on the day it was announced (23 February 2024), and FDA understands that Oncopeptides does not currently market Pepaxto in the US.In accordance with the amended procedures for withdrawal of accelerated approval that were enacted in 2023, 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. …