Melphalan Flufenamide (Melflufen)




Melphalan Flufenamide (Melflufen, PEPAXTO®) is a peptide-drug conjugate (PDC) that uses specific enzymes that are produced in large amounts by myeloma cells. Once the drug enters the cell, it delivers a toxic payload to attack the cancer cell.

September 22, 2022: In a 14 to 2 vote, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted that the benefit-risk profile of melphalan flufenamide (Pepaxto) is not favorable for the approved indicated population of patients with relapsed/refractory multiple myeloma. Learn more

SparkCures ID 264
Developed By Oncopeptides AB
Brand Name Pepaxto®
Generic Name Melphalan Flufenamide (Melflufen)
Treatment Classifications
  • Peptide-Conjugated Alkylator

Clinical Trials

Published Results

Full data set of Oncopeptides phase 2 HORIZON study in multiple myeloma published in the Journal of Clinical Oncology

December 09, 2020

The phase 2 HORIZON data are the basis for the ongoing priority review of the New Drug Application to the US Food and Drug Administration FDA, for accelerated approval of melflufen in combination with dexamethasone in triple-class refractory multiple myeloma patients, who are refractory to at least one proteasome inhibitor, one immunomodulatory drug and one anti-CD38 monoclonal antibody.

Oncopeptides AB


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