Melphalan flufenamide hydrochlorideProduct ingredient for Melphalan flufenamide

Melphalan flufenamide, also known as melflufen or J1, is a prodrug of melphalan.^1,9 Melphalan flufenamide is more readily uptaken by cells than melphalan, and is cleaved to the active metabolite by aminopeptidases.¹ In vitro models show that melphalan is 10 to hundreds of times more potent than melphalan.¹ The increased potency makes melphalan flufenamide a treatment option for patients with relapsed or refractory multiple myeloma who have attempted at least 4 lines of therapy already.^2,9

Melphalan flufenamide was granted FDA approval on 26 February 2021.⁹. It has since been withdrawn from the market in the wake of the phase 3 OCEAN trial which showed a decrease in overall survival in comparison to standard treatment with pomalidomide and dexamethasone despite superior progression-free survival.^10,11

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