Pepaxto (melphalan flufenamide)

Pepaxto may cause serious side effects, including:

• Low blood cell counts are common with Pepaxto and can be serious. Your healthcare provider will do blood tests as needed to check your blood cell counts during your treatment with Pepaxto.
  • Low platelet counts: Tell your healthcare provider right away if you have a bleeding or bruising under the skin.
  • Low red blood cell counts: Tell your healthcare provider if you are feeling weak, tired or you get tired easily, you look pale, or if you feel short of breath.
  • Low white blood cell counts: A low white blood cell count increases the risk of infections.
• Infections. Pepaxto can cause infections that have led to death. Tell your healthcareprovider right away if you develop new or worsening signs or symptoms of infection such as fever, chills, cough, pain, or burning during urination during treatment with Pepaxto.
• Secondary cancers. New cancers such as myelodysplastic syndromes or acute leukemia have happened in people with multiple myeloma who have received Pepaxto. Your healthcare provider will monitor you for new cancers.

Your healthcare provider may change your dose of Pepaxto, stop your treatment for a period of time, or completely stop your treatment if you have certain side effects.

Pepaxto may cause fertility problems in males and females, which may affect your ability to have children. Talk with your healthcare provider if you have concerns about fertility.

The most common side effects of Pepaxto include, low blood cell counts, fatigue, nausea, diarrhea, fever, and cold-like symptoms (respiratory tract infection).

These are not all of the possible side effects of Pepaxto.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Recommended Dosage

• The recommended dosage of Pepaxto is 40 mg administered intravenously over 30 minutes on Day 1 of each 28-day cycle until disease progression or until unacceptable toxicity. Administer dexamethasone 40 mg orally or intravenously on Days 1, 8, 15 and 22 of each cycle.
• For patients 75 years of age or older, reduce the dose of dexamethasone to 20 mg.
• Refer to the prescribing information for dexamethasone for additional dosing information.

Recommended Premedication And Concomitant Medications

• Consider providing a serotonin-3 (5-HT3) receptor antagonist or other antiemetics prior to and during the treatment with Pepaxto.

Dosage Modifications For Adverse Reactions

• Withold Pepaxto if the neutrophil count is less than 1 x 10⁹/L or the platelet count is less than 50 x 10⁹/L.
• The recommended dose reductions and dosage modifications for adverse reactions for Pepaxto are presented in Table 1 and Table 2, respectively.

Table 1: Recommended Dose Reductions for Adverse Reactions of Pepaxto

Table 2: Recommended Dosage Modifications for Adverse Reactions of Pepaxto

• Based on its mechanism of action, Pepaxto can cause fetal harm when administered to a pregnant woman.
• There are no available data on Pepaxto use in pregnant women to evaluate for a drug-associated risk. Pepaxto is a genotoxic drug. Advise pregnant women of the potential risk to a fetus.
• There is no data on the presence of melphalan flufenamide or its metabolites in human breast milk, or the effects on the breastfed child or on milk production.
• Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with Pepaxto and for 1 week after the last dose.