Melphalan Flufenamide Receives Accelerated Approval for Resistant Multiple Myeloma
Posted: Tuesday, March 2, 2021
On February 26, the U.S. Food and Drug Administration (FDA) moved from Priority Review to accelerated approval for melphalan flufenamide (Pepaxto) in combination with dexamethasone. The combination treatment is indicated for adults with relapsed or refractory multiple myeloma who had 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.
The FDA approval was based on the multicenter, single-arm HORIZON clinical trial. Patients with relapsed or refractory multiple myeloma were eligible for enrollment. Each patient was administered 40 mg of intravenous melphalan flufenamide on day 1 and 40 mg of oral dexamethasone or 20 mg of dexamethasone if older than age 75 on days 1, 8, 15, and 22 of a 28-day cycle until disease progression or unacceptable toxicity.
The overall response rate in a subpopulation of 97 patients was 23.7%. The median duration of response was 4.2 months.
The most common adverse reactions in 20% or more of patients receiving melphalan flufenamide included fatigue, nausea, diarrhea, pyrexia, and respiratory tract infection. The most common laboratory abnormalities in 50% or more of patients were decreased leukocytes, platelets, lymphocytes, neutrophils, hemoglobin levels, and increased creatinine.
The recommended dose of melphalan flufenamide in combination with dexamethasone is 40 mg intravenously over 30 minutes on day 1 of each 28-day treatment cycle. For full prescribing information, visit accessdata.fda.gov.
