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Pepaxto FDA Approval History

Last updated by Judith Stewart, BPharm on Feb 26, 2024.

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 following consultation with the U.S. FDA regarding the results of the Phase 3 OCEAN study.
On February 23, 2024, the FDA announced its final decision to withdraw approval of Pepaxto. The agency determined the following grounds for withdrawal were met:
  1. the confirmatory study conducted as a condition of accelerated approval did not confirm Pepaxto’s clinical benefit, and
  2. the available evidence demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use.

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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