The words approved in red capitalized letters.

Oncopeptides obtains FDA nod for Pepaxto

Sandra Levy
Senior Editor
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Oncopeptides has received the Food and Drug Administration’s blessing for Pepaxto (melphalan flufenamide), known during clinical development as melflufen, in combination with dexamethasone.

The medication is used for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.

Pepaxto is the first anticancer peptide-drug conjugate approved in multiple myeloma.

"While the treatment landscape for multiple myeloma has dramatically improved in recent years, once patients become resistant to existing classes of therapy they can face a very guarded prognosis," said Paul Richardson, clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute. "Research has shown melphalan flufenamide to be a novel and innovative therapeutic option, which is active in refractory disease and has manageable toxicity, with the convenience of being administered by infusion once a month. Based on our findings, melphalan flufenamide is an important addition to the treatment armamentarium, with the potential to meaningfully improve outcomes in an area of important unmet medical need."

"We are proud to bring forward the first anticancer peptide-drug conjugate approved by the FDA for multiple myeloma," said Jakob Lindberg, chief scientific officer at Oncopeptides. "Pepaxto uses innovative technology that links a peptide carrier to a cytotoxic agent. The conjugated agent is a highly lipophilic compound, which allows it to be rapidly distributed into cells. The compound then leverages amino peptidases that are overexpressed in Multiple Myeloma cells, causing the release of the cytotoxic payload."

Marty Duvall, Oncopeptides CEO said, "The accelerated U.S. approval of Pepaxto is a key step forward in fulfilling Oncopeptides' core mission, to bring hope to patients in their battle against difficult-to-treat hematological diseases. Moving ahead, our focus is to further advance Pepaxtyo.  We look forward to receiving top line data from the phase 3 OCEAN study in the second quarter.  This comparative study is designed to support a future supplementary New Drug Application to expand the label."

Oncopeptides expects Pepaxto to become commercially available in the United States within approximately two weeks.