melphalan flufenamide + dexamethasone

**Melphalan flufenamide + dexamethasone** is a combination therapy consisting of melphalan flufenamide, a first-in-class peptide-drug conjugate delivering alkylating agents directly into tumor cells via aminopeptidase-mediated cleavage, and dexamethasone, a synthetic glucocorticoid. The combination was investigated and approved for use in relapsed and refractory multiple myeloma (RRMM) in patients who had received multiple prior therapies, including immunomodulatory agents and proteasome inhibitors. Melphalan flufenamide acts by inducing DNA damage via alkylation upon release in myeloma cells, while dexamethasone exerts anti-inflammatory and anti-myeloma effects via glucocorticoid receptor agonism. Initial accelerated approval was based on clinical benefit in highly pretreated RRMM populations; however, subsequent post-marketing studies led to product withdrawal due to lack of confirmatory benefit and safety concerns.

Brand names

Pepaxto + dexamethasone

Other names

melphalan flufenamide + dexamethasonemelflufen + dexamethasone