Melphalan flufenamide

Melphalan flufenamide, sold under the brand names Pepaxto and Pepaxti, is a medication for treating refractory multiple myeloma, available in multiple European countries but not in the United States. It is an alkylating agent that functions as a peptidase enhanced cytotoxic, delivering the alkylating agent melphalan in a targeted way within tumor cells. It is given intravenously. In the United States before its withdrawal it was indicated for adults with relapsed or refractory multiple myeloma who had received at least four prior lines of therapy and were refractory to proteasome inhibitors, immunomodulatory agents and CD-38 monoclonal antibodies, while the European Union indication requires at least three prior therapy lines. It received FDA approval in February 2021, was withdrawn from the United States market in October 2021, and received European Union approval in August 2022.