FDA Brief: Priority Review for Melphalan Flufenamide Combination in Myeloma
Posted: Tuesday, September 8, 2020
On August 29, the U.S. Food and Drug Administration (FDA) granted Priority Review for melphalan flufenamide (Melflufen) in combination with dexamethasone for the treatment of adults with triple-class–refractory multiple myeloma whose disease is resistant to at least one proteasome inhibitor, one immunomodulatory agent, and one anti–CD-38 monoclonal antibody. Melphalan flufenamide is a first-in-class peptide-drug conjugate that targets aminopeptidases and rapidly releases alkylating agents into tumor cells.
The Priority Review was based on data from the phase II HORIZON study, which evaluated intravenous melphalan flufenamide in combination with dexamethasone in patients with relapsed or refractory multiple myeloma. The results showed “encouraging” efficacy and a clinically manageable safety profile in heavily pretreated patients. However, researchers primarily reported hematologic adverse events along with a low incidence of nonhematologic adverse events.