Is Melphalan flufenamide approved by the FDA?

Melphalan flufenamide, marketed under the brand name Pepaxto, is an alkylating agent used in oncology. Melphalan flufenamide (Pepaxto) was approved by the U.S. Food and Drug Administration (FDA) on February 26, 2021. The approval was based on clinical trial data demonstrating its efficacy in patients with relapsed or refractory multiple myeloma who had undergone extensive prior treatment.

Uses and Administration

Uses:

• Melphalan flufenamide is used in combination with dexamethasone to treat multiple myeloma in patients whose disease has returned and who have received at least four prior treatments that were not effective.

Administration:

• The medication is administered intravenously by a healthcare professional. It is given through a vein, usually once every 28 days, and the infusion takes at least 30 minutes to complete.

Precautions and Considerations

Before Using Melphalan Flufenamide:

• Patients should inform their doctor of any allergies, as well as any other medical conditions, especially if they have had anemia, neutropenia, thrombocytopenia, or infections.
• The safety and efficacy in pediatric populations have not been established.
• Geriatric patients may use this medication without specific age-related concerns, but close monitoring is essential.

Potential Side Effects:

• Serious Side Effects: Back, arm, or leg pain; black, tarry stools; bleeding gums; blood in the urine or stools; bone pain; chest pain or tightness; constipation; decreased appetite; decreased urine; diarrhea; fever or chills; lower back or side pain; nausea; numbness or tingling; pain in the joints; pale skin; seizures; sleeplessness; sore throat; unusual bleeding or bruising; unusual tiredness or weakness; vomiting.
• Common Side Effects: Skin itching, rash, or redness; swelling of the face, throat, or tongue; trouble sleeping.

Patients should report any side effects to their healthcare provider or directly to the FDA at 1-800-FDA-1088.

Interactions

Drug Interactions:

• Melphalan flufenamide may interact with other medications, including over-the-counter drugs and supplements. Patients should provide a complete list of their current medications to their healthcare provider.

Food/Tobacco/Alcohol Interactions:

• Patients should discuss with their healthcare professional the use of food, alcohol, or tobacco, as these can affect the efficacy and safety of the treatment.

Detailed Dosage Information

Usual Adult Dose for Multiple Myeloma:

• The recommended dose is 40 mg administered as an intravenous infusion once every 28 days, in combination with dexamethasone.

Missed Dose:

• If a dose is missed, patients should contact their healthcare provider for instructions.

Conclusion

It offers a new therapeutic option for this patient population, though it comes with significant potential side effects and precautions. As always, patients should have thorough discussions with their healthcare providers to weigh the benefits and risks of this treatment.

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