The agency granted accelerated approval to Pepaxto in combination with dexamethasone in adult patients with relapsed or refractory multiple myeloma, who received at least four prior lines of therapy.
The Food and Drug Administration (FDA) granted accelerated approval to Pepaxto (melphalan flufenamide) in combination with dexamethasone in adult patients with relapsed or refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one CD38-directed monoclonal antibody, according to Oncopeptides AB, the agent’s manufacturer.
Of note, the agent is the first anticancer peptide-drug conjugate approved by the FDA.
“Melphalan flufenamide is a novel and innovative therapeutic option which is active in patients with multiple myeloma who have a refractory disease, and the product has a manageable toxicity,” Dr. Ola Landgren, chief of the Myeloma Program and leader of experimental therapeutics program in the Division of Hematology at Sylvester Comprehensive Cancer Center at the University of Miami Health System, said in a press release. “Melphalan flufenamide will complement existing treatment regimens and contribute to address the growing unmet medical need among patients with relapsed or refractory multiple myeloma”.
The agency based its decision on data from the phase 2 HORIZON trial, designed to evaluate intravenous Pepaxto plus dexamethasone in 157 patients with relapsed/refractory multiple myeloma.
The combination demonstrated an overall response rate of 23.7%, with a median duration of response of 4.2 months.
Looking ahead, Marty J Duvall, chief executive officer at Oncopeptides AB, noted the company will be receiving top line data from the phase 3 OCEAN-study, a trial investigating Pepaxto versus pomalidomide in patients with relapsed/refractory multiple myeloma “to support a future supplementary new drug application to expand the label.”