Melphalan Flufenamide
Melphalan Flufenamide is a peptide-conjugated alkylating agent developed as a prodrug of melphalan for the treatment of relapsed or refractory multiple myeloma. It works by delivering alkylating activity to cancer cells through enhanced cellular uptake due to its lipophilic properties. The drug was specifically designed for patients who had received at least two prior lines of therapy.
Melphalan Flufenamide is an alkylating chemotherapy agent used to treat relapsed or refractory multiple myeloma in patients who have received prior therapies.
Key facts at a glance
- ATC
- L01AA10
- RxNorm
- 2531369
- Generic
- Melphalan Flufenamide
- Class
- atc-l01
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
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Treatment of relapsed or refractory multiple myeloma in adult patients who have received at least two prior lines of therapy, including lenalidomide and a proteasome inhibitor, used in combination with dexamethasone.
Melphalan Flufenamide is a lipophilic prodrug that rapidly enters cells where peptidases cleave it to release melphalan, an alkylating agent. The alkylating moiety binds to DNA, causing cross-linking that inhibits DNA replication and leads to cancer cell death.
Administered as an intravenous infusion. Associated with significant myelosuppression, particularly thrombocytopenia and neutropenia. May cause hepatotoxicity. The drug was withdrawn from the US market in 2022 due to failure to demonstrate efficacy in a confirmatory trial. Use requires careful patient selection and monitoring of blood counts.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 25, 2026. · Read our editorial policy