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:
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:
- the confirmatory study conducted as a condition of accelerated approval did not confirm Pepaxto’s clinical benefit, and
- the available evidence demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use.
- 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
Further information
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