Update: 7/28/21 - FDA alerts patients and health care professionals about clinical trial results showing an increased risk of death associated with Pepaxto (melphalan flufenamide)


What Is Pepaxto?

Pepaxto® is an alkylating drug approved by the U.S. Food and Drug Administration (FDA) in combination with the steroid dexamethasone for the treatment of adult patients with relapsed or refractory myeloma. Pepaxto is the first anticancer peptide-drug conjugate approved by the FDA.

Who Is a Candidate for Pepaxto?

Myeloma patients with relapsed or refractory disease who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor (e.g., Velcade® [bortezomib], Kyprolis® [carfilzomib], or Ninlaro® [ixazomib]), one immunomodulatory drug (e.g., Revlimid® [lenalidomide], Pomalyst® [pomalidomide], or thalidomide), and one CD38-directed monoclonal antibody (e.g., Darzalex® [daratumumab] or Sarclisa® [isatuximab]).

Pepaxto is not recommended for use as a conditioning regimen for transplant.

Pepaxto is contraindicated for patients with a history of serious hypersensitivity reaction to melphalan flufenamide or melphalan.

How Does Pepaxto Work?

When Pepaxto enters the myeloma cell, it is released and then damages the cell from the inside.

How Is Pepaxto Given?

Pepaxto is given intravenously via a central line at a dose of 40 mg over 30 minutes on Day 1 of each 28-day treatment cycle. Pepaxto is given in combination with dexamethasone. 

Warnings and Precautions

Your doctor will monitor your blood counts at baseline, during treatment, and as clinically indicated. Dose delay or reduction may be required if you experience thrombocytopenia (low count of platelets), neutropenia (low count of neutrophils), anemia (low count of red blood cells), infections, and/or secondary malignancies.

Pepaxto can cause harm to an unborn baby. Females of reproductive potential and males with female partners of reproductive potential must use effective birth control during treatment with Pepaxto and for 30 days after the last dose for women and for 90 days after the last dose for men. Patients taking Pepaxto are advised not to breastfeed.

Possible Side Effects of Pepaxto

Most common side effects occurring in more than 20% of patients are fatigue, nausea, diarrhea, fever, and respiratory tract infection.

Most common laboratory abnormalities occurring in 50% or more of patients are a decrease in leukocytes, platelets, lymphocytes, neutrophils, and an increase in creatinine.


Your doctor is always your primary resource for medical advice. To access the ON COURSE support resources for patient and caregiver from Oncopeptides, the maker of Pepaxto, please visit or call 1-844-300-ONCO (1-844-300-6626).

As always, the IMF urges you to discuss all medical issues with your doctor, and to contact the IMF InfoLine with your myeloma questions and concerns. 



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