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Oncology · FDA Approved

Melphalan Flufenamide (Pepaxto)

FDA-approved

How to ask your doctor
Written by Peptide Association Editorial TeamLast reviewed Editorial policy

01 · Overview

Melphalan flufenamide (melflufen, Pepaxto) is a first-in-class peptide-drug conjugate that links the alkylating agent melphalan to a dipeptide carrier (PMID 33296242). It targets aminopeptidases in tumor cells, where it is hydrolyzed to release alkylating molecules that become trapped inside the cell (PMID 32992506).

The aim is to increase tumor exposure to the alkylator while improving the therapeutic index of cytotoxic chemotherapy. It received FDA accelerated approval for relapsed or refractory multiple myeloma but has since been withdrawn from the US market.

02 · Research summary & key findings

Melflufen received FDA accelerated approval in early 2021 for heavily pretreated relapsed or refractory multiple myeloma (PMID 35983927), based on the single-arm phase 2 HORIZON trial, in which the overall response rate was 29% in 157 patients (PMID 33296242). In the confirmatory phase 3 OCEAN trial, melflufen improved progression-free survival over pomalidomide (6.8 vs 4.9 months) but median overall survival was numerically shorter (19.8 vs 25.0 months, not statistically significant) (PMID 35032434). It was withdrawn from the US market in October 2021 after the OCEAN results (PMID 35196605).

03 · Regulatory status

Melphalan Flufenamide (Pepaxto) has received FDA approval and is available for clinical use under appropriate medical supervision. Consult a qualified healthcare provider for prescribing information.

Melphalan Flufenamide (Pepaxto) is an FDA-approved medicine prescribed for the conditions on its label. It is not part of our intake, so we do not propose protocols for it. If you think it may be relevant to you, talk to your own doctor. How to ask your doctor.

04 · Administration routes

  • Intravenous

References

  1. [1]Melflufen: A Peptide-Drug Conjugate for the Treatment of Multiple Myeloma J Clin Med, 2020. PMID 32992506
  2. [2]Melflufen and Dexamethasone in Heavily Pretreated Relapsed and Refractory Multiple Myeloma J Clin Oncol, 2021. PMID 33296242
  3. [3]Melflufen or pomalidomide plus dexamethasone for patients with multiple myeloma refractory to lenalidomide (OCEAN): a randomised, head-to-head, open-label, phase 3 study Lancet Haematol, 2022. PMID 35032434
  4. [4]The approval and withdrawal of melphalan flufenamide (melflufen): Implications for the state of the FDA Transl Oncol, 2022. PMID 35196605
  5. [5]Melphalan flufenamide for relapsed/refractory multiple myeloma Drugs Today (Barc), 2022. PMID 35983927

Primary literature

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Disclaimer: This information is provided for educational and research purposes only. It is not intended as medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before starting any peptide therapy. The Peptide Association does not endorse or recommend any specific treatment protocol. See our editorial policy for how this content is produced and reviewed. Clinicians: join the network.