OncologyIn Clinical Trials

Melphalan Flufenamide (Pepaxto)

Intravenous

Overview

Melphalan flufenamide is a first-in-class peptide-drug conjugate that links the alkylating agent melphalan to a dipeptide carrier. The dipeptide facilitates cellular uptake via aminopeptidases that are overexpressed in myeloma cells, where intracellular cleavage releases active melphalan.

Mechanism of Action

This targeted delivery aims to enhance tumor exposure while reducing systemic toxicity. The compound represents a prodrug strategy to improve the therapeutic index of cytotoxic chemotherapy..

Research Summary & Key Findings

Accelerated FDA approval was granted in February 2021 based on the single-arm HORIZON trial in relapsed/refractory multiple myeloma (ORR 29%). The confirmatory OCEAN trial subsequently showed inferior overall survival compared to standard regimens, leading to voluntary market withdrawal in October 2021. This case highlighted challenges in confirmatory trial design for accelerated approvals.

Clinical Status

In Clinical Trials

Melphalan Flufenamide (Pepaxto) is currently undergoing clinical trials. It is not yet FDA-approved and is available only through clinical research settings or investigational protocols.

Administration Routes

Intravenous

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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.