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Class: Chemotherapy
Generic Name: melphalan flufenamide
Trade Name: Pepaxto®

For which conditions is Pepaxto approved for? Pepaxto is approved in combination with dexamethasone for the treatment of adults with multiple myeloma who have had at least four prior treatments and have not responded or stopped responding to treatment. The four prior treatments must include a proteasome inhibitor, immunotherapy and on CD38-directed antibody. Pepaxto is not appropriate as pre-transplant treatment.

What is the mechanism of action? Pepaxto is classified as an alkylating agent. An alkylating agent produces its anti-cancer effects by causing a chemical reaction that damages the DNA in a cell. The DNA damage caused by Pepaxto results in cell death.

How is Pepaxto typically given (administered)? Pepaxto is given intravenously (into a vein), typically once every four weeks.

How are patients typically monitored? Patients will usually have scheduled meetings with their healthcare provider while they are being treated with Pepaxto. Typically, blood will be drawn to check levels of blood cells and to monitor functions of some organ systems, such as the kidneys or liver. Patients may also undergo physical examinations, scans or other measures to assess side effects and response to therapy.

What are the common side effects of treatment with Pepaxto?

  • Low levels of white blood cells – increases the risk of infection
  • Low levels of red blood cells – increases the risk of anemia and blood transfusions
  • Low levels of platelets – increases the risk of bleeding
  • Fatigue Nausea Diarrhea Fever
  • Cold-like symptoms

This is not a complete list of side effects. Some patients may experience other side effects that are not listed here such as weight gain, decreased sex drive and erectile function problems. Patients may wish to discuss with their physician the other less common side effects of this drug, some of which may be serious.

Some side effects may require medical attention. Other side effects do not require medical attention and may go away during treatment. Patients should check with their physician about any side effects that continue or are bothersome.

What can patients do to help alleviate or prevent discomfort and side effects?

  • Pay careful attention to the physician’s instructions and inform the physician of any side effects.
  • If you have a history of severe allergic reaction to melphalan flufenamide or melphalan, do not take Pepaxto.
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Are there any special precautions patients should be aware of before starting treatment?

  • Patients should inform their physician about all medical conditions.
  • Patients should inform their physician of any other medication or supplement they are taking (whether prescription or over-the-counter).
  • Talk to your doctor if your partner is pregnant or planning to become pregnant. If a man’s sexual partner is not pregnant but could become pregnant, effective birth control should be used during treatment and for three months after treatment ends. Females should use an effective method of birth control during treatment and for 6 months after the last does of Pepaxto.
  • Do not breastfeed during treatment or for 1 week after the last dose of Pepaxto.

When should patients notify their physician?

Tell your doctor if you experience any side effects that bother you or don’t go away. Pepaxto may cause serious side effects including infections that have led to death. Tell your healthcare provider right away if you experience:

  • Symptoms of low blood cell counts including bleeding, bruising under the skin, feeling of weakness or fatigue, looking pale, shortness of breath.
  • Symptoms of infections including fever, chills, cough, pain or burning during urination.

What is a package insert?
A package insert is required by the FDA and contains a summary of the essential scientific information needed for the safe and effective use of the drug for healthcare providers and consumers. A package insert typically includes information regarding specific indications, administration schedules, dosing, side effects, contraindications, results from some clinical trials, chemical structure, pharmacokinetics and metabolism of the specific drug. By carefully reviewing the package insert, you will get the most complete and current information about how to safely use this drug. If you do not have the package insert for the drug you are using, your pharmacist or physician may be able to provide you with a copy.

Last updated 04/21

Important Limitations of Use
The information provided above on the drug you have selected is provided for your information only and is not a substitute for consultation with an appropriate medical doctor. We are providing this information solely as a courtesy and, as such, it is in no way a recommendation as to the safety, efficacy or appropriateness of any particular drug, regimen, dosing schedule for any particular cancer, condition or patient nor is it in any way to be considered medical advice. Patients should discuss the appropriateness of a particular drug or chemotherapy regimen with their physician.

As with any printed reference, the use of particular drugs, regimens and drug dosages may become out-of-date over time, since new information may have been published and become generally accepted after the latest update to this printed information. Please keep in mind that health care professionals are fully responsible for practicing within current standards, avoiding use of outdated regimens, employing good clinical judgment in selecting drugs and/or regimens, in calculating doses for individual patients, and verifying all dosage calculations.



The prescribing physician is solely responsible for making all decisions relating to appropriate patient care including, but not limited to, drugs, regimens, dose, schedule, and any supportive care.