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Melphalan flufenamide

Generic name: melphalan flufenamide [ MEL-fa-lan-floo-FEN-a-mide ]
Brand name: Pepaxto
Dosage form: intravenous powder for injection (20 mg)
Drug class: Alkylating agents

Medically reviewed by on Apr 14, 2021. Written by Cerner Multum.

What is melphalan flufenamide?

Melphalan flufenamide is used together with dexamethasone (a steroid medicine) to treat adults with multiple myeloma.

Melphalan flufenamide is given after at least four other cancer treatments did not work or have stopped working.

Melphalan flufenamide may also be used for purposes not listed in this medication guide.


Melphalan flufenamide can increase your risk of bleeding or infection. Call your doctor if you have unusual bruising or bleeding, or new signs of infection (fever, chills, cough, burning when you urinate).

Before taking this medicine

You should not be treated with melphalan flufenamide if you are allergic to it.

Tell your doctor if you have any type of infection.

Using melphalan flufenamide may increase your risk of developing other cancers, such as leukemia. Ask your doctor about this risk.

You may need to have a negative pregnancy test before starting this treatment.

Melphalan flufenamide can harm an unborn baby if the mother or the father is using melphalan flufenamide.

  • If you are a woman, do not use melphalan flufenamide if you are pregnant. Use effective birth control to prevent pregnancy while you are using this medicine and for at least 6 months after your last dose.

  • If you are a man, use effective birth control if your sex partner is able to get pregnant. Keep using birth control for at least 3 months after your last dose.

  • Tell your doctor right away if a pregnancy occurs while either the mother or the father is using melphalan flufenamide.

Pregnancy may be less likely to occur while the mother or the father is using this medicine. Both men and women should still use birth control to prevent pregnancy because the medicine can harm an unborn baby.

Do not breastfeed while using this medicine, and for at least 1 week after your last dose.

How is melphalan flufenamide given?

Melphalan flufenamide is given as an infusion into a vein. A healthcare provider will give you this injection.

This medicine is given in a 28-day treatment cycle. You will receive melphalan flufenamide only on the first day of each cycle. Melphalan flufenamide must be given slowly, and the infusion can take about 30 minutes to complete.

Dexamethasone is either taken by mouth or given as an injection given by your healthcare professional. Usually, the dexamethasone is used every 7 days of each treatment cycle.

Your doctor will determine how long to treat you with melphalan flufenamide and dexamethasone. Follow your doctor's dosing instructions very carefully.

You may be given other medicine to help prevent nausea. Keep using this medicine for as long as your doctor has prescribed.

You may need frequent medical tests to be sure this medicine is not causing harmful effects. Your cancer treatments may be delayed or stopped based on the results.

What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your melphalan flufenamide injection.

What happens if I overdose?

Since melphalan flufenamide is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid while receiving melphalan flufenamide?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Melphalan flufenamide side effects

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Melphalan flufenamide may cause serious side effects. Call your doctor at once if you have:

  • a new or worsening cough;

  • pain or burning when you urinate; or

  • low blood cell counts--fever, chills, tiredness, mouth sores, skin sores, easy bruising, unusual bleeding, pale skin, cold hands and feet, feeling light-headed or short of breath.

Common side effects of melphalan flufenamide may include:

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Melphalan flufenamide dosing information

Usual Adult Dose for Multiple Myeloma:

40 mg IV over 30-minute on Day 1 of each 28-day cycle

-This drug is not recommended for use as a conditioning regimen for transplant outside controlled clinical trials.
-Continue treatment until disease progression or unacceptable toxicity.
-Administer dexamethasone 40 mg orally or IV on Days 1, 8, 15, and 22 of each cycle.
-Dexamethasone dose should be reduced to 20 mg in patients 75 years of age or older.
-Consider the use of antiemetic medicine such as a serotonin-3 receptor antagonist or other antiemetics prior to and during treatment.

-In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma 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

What other drugs will affect melphalan flufenamide?

Other drugs may affect melphalan flufenamide, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

More about melphalan flufenamide

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Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.