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Melphalan flufenamide (Intravenous)

Medically reviewed by Last updated on Jul 29, 2022.

Commonly used brand name(s)

In the U.S.

  • Pepaxto

Pharmacologic Class: Alkylating Agent

Chemical Class: Nitrogen Mustard

Uses for melphalan flufenamide

Melphalan flufenamide injection is used in combination with dexamethasone to treat multiple myeloma (bone marrow cancer) in patients whose cancer has come back and who have received at least 4 previous treatments (eg, a CD38-directed monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent) that did not work well.

Melphalan flufenamide belongs to the group of medicines called alkylating agents (cancer medicines). It works by interfering with the growth of cancer cells, which are eventually destroyed by the body.

Melphalan flufenamide is to be given only by or under the supervision of your doctor.

Before using melphalan flufenamide

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For melphalan flufenamide, the following should be considered:


Tell your doctor if you have ever had any unusual or allergic reaction to melphalan flufenamide or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.


Appropriate studies have not been performed on the relationship of age to the effects of melphalan flufenamide injection in the pediatric population. Safety and efficacy have not been established.


Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of melphalan flufenamide injection in the elderly.

Interactions with medicines

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Interactions with food/tobacco/alcohol

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems

The presence of other medical problems may affect the use of melphalan flufenamide. Make sure you tell your doctor if you have any other medical problems, especially:

  • Anemia or
  • Neutropenia (low number of white blood cells) or
  • Thrombocytopenia (low number of platelets)—Use with caution. May make these conditions worse.
  • Infection—May decrease your ability to fight an infection.

Proper use of melphalan flufenamide

A nurse or other trained health professional will give you melphalan flufenamide in a hospital. It is given through a needle placed into one of your veins. Melphalan flufenamide must be given slowly, so the IV tube will need to stay in place for at least 30 minutes. It is usually given one time for 28 days.

Melphalan flufenamide should come with a patient information leaflet. Read and follow these instructions carefully. Ask your doctor or pharmacist if you have any questions.

You may receive medicines to help prevent nausea before and during treatment with melphalan flufenamide.

Precautions while using melphalan flufenamide

It is very important that your doctor check your progress closely while you are receiving melphalan flufenamide to make sure that the medicine is working properly. Blood tests may be needed to check for unwanted effects.

Receiving melphalan flufenamide while you are pregnant can harm your unborn baby. If you are a woman who can bear children, your doctor may give you a pregnancy test before you start using melphalan flufenamide to make sure you are not pregnant. Use an effective form of birth control while you are receiving melphalan flufenamide and for at least 6 months after the last dose to keep from getting pregnant. Males who are receiving melphalan flufenamide, with female partners who can become pregnant, must use effective birth control during and for at least 3 months after the last dose of melphalan flufenamide. If you think you have become pregnant while receiving the medicine, tell your doctor right away.

Some men and women who use melphalan flufenamide have become infertile (unable to have children). Talk with your doctor before using melphalan flufenamide if you plan to have children.

Melphalan flufenamide can temporarily lower the number of white blood cells in your blood, increasing the chance of getting an infection. It can also lower the number of platelets, which are necessary for proper blood clotting. If this occurs, there are certain precautions you can take, especially when your blood count is low, to reduce the risk of infection or bleeding:

  • If you can, avoid people with infections. Check with your doctor immediately if you think you are getting an infection or if you get a fever or chills, cough or hoarseness, lower back or side pain, or painful or difficult urination.
  • Check with your doctor immediately if you notice any unusual bleeding or bruising, black, tarry stools, blood in the urine or stools, or pinpoint red spots on your skin.
  • Be careful when using a regular toothbrush, dental floss, or toothpick. Your medical doctor, dentist, or nurse may recommend other ways to clean your teeth and gums. Check with your medical doctor before having any dental work done.
  • Do not touch your eyes or the inside of your nose unless you have just washed your hands and have not touched anything else in the meantime.
  • Be careful not to cut yourself when you are using sharp objects such as a safety razor or fingernail or toenail cutters.
  • Avoid contact sports or other situations where bruising or injury could occur.

Melphalan flufenamide may cause serious infections (eg, respiratory tract infection, pneumonia, sepsis). Check with your doctor right away if you have chest pain, chills, confusion, cough, dizziness or lightheadedness, fainting, fast heartbeat, fever, sneezing, sore throat, tightness in the chest, or trouble breathing.

Using melphalan flufenamide may increase your risk of getting other types of cancer (eg, myelodysplastic syndromes, acute leukemia). Talk to your doctor if you have concerns about this risk.

Melphalan flufenamide side effects

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

  • Back, arm, or leg pain
  • black, tarry stools
  • bleeding gums
  • blood in the urine or stools
  • bone pain
  • chest pain or tightness
  • constipation
  • cough
  • decreased appetite
  • decreased urine
  • diarrhea
  • difficulty in moving
  • dry mouth
  • fever or chills
  • increased thirst
  • loss of appetite
  • lower back or side pain
  • mood or mental changes
  • muscle pain, cramps, or stiffness
  • nausea
  • numbness or tingling in the hands, feet, or lips
  • pain in the joints
  • painful or difficult urination
  • pale skin
  • pinpoint red spots on the skin
  • seizures
  • sleeplessness
  • sneezing
  • sore throat
  • stomach cramps
  • swelling of the hands, ankles, feet, or lower legs
  • tremor
  • trouble breathing
  • ulcers, sores, or white spots in the mouth
  • unusual bleeding or bruising
  • unusual tiredness or weakness
  • vomiting

Less common

  • Confusion
  • coughing up blood
  • difficulty in swallowing
  • dizziness
  • fainting
  • fast heartbeat
  • headache
  • increased menstrual flow or vaginal bleeding
  • lightheadedness
  • nosebleeds
  • paralysis
  • prolonged bleeding from cuts
  • red or dark brown urine
  • red or black, tarry stools

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

  • Skin itching, rash, or redness
  • swelling of the face, throat, or tongue
  • trouble sleeping

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

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

Further information

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