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Pepaxto FDA Approval History

Last updated by Judith Stewart, BPharm on April 4, 2021.

FDA Approved: Yes (Discontinued) (First approved February 26, 2021)
Brand name: Pepaxto
Generic name: melphalan flufenamide
Dosage form: Injection
Company: Oncopeptides AB
Treatment for: Multiple Myeloma

Pepaxto (melphalan flufenamide) is an anticancer peptide-drug conjugate indicated for use in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma.

On October 22, 2021, Oncopeptides announced the decision to voluntarily withdraw Pepaxto from the U.S. market. The decision was made following consultation with the U.S. FDA regarding the results of the Phase 3 OCEAN study, which showed an overall survival in the intent-to-treat population with a hazard ratio of 1.104.
  • Pepaxto is used in patients 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.
  • Pepaxto is administered intravenously over 30 minutes on Day 1 of each 28-day treatment cycle, in combination with dexamethasone.
  • Pepaxto can cause serious adverse reactions including thrombocytopenia, neutropenia, anemia, infections, secondary malignancies, and embryo-fetal toxicity.
  • Common adverse reactions include fatigue, nausea, diarrhea, pyrexia, respiratory tract infection, and laboratory abnormalities.

Development timeline for Pepaxto

Feb 26, 2021Approval FDA Approves Pepaxto (melphalan flufenamide) for Patients with Relapsed or Refractory Multiple Myeloma
Dec  7, 2019Oncopeptides Presents Updated Efficacy and Safety Data from Melflufen Pivotal Phase 2 HORIZON Study in Patients With RRMM at ASH Annual Meeting 2019

Further information

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