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FDA Approves Melphalan Flufenamide for Relapsed/Refractory MM
On February 26, 2021, the FDA approved melphalan flufenamide (Pepaxto; Oncopeptides AB) for use in combination with dexamethasone in the treatment of patients with relapsed or refractory multiple myeloma (MM) who received at least 4 prior lines of therapy and whose disease is refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent, and 1 CD-38 directed monoclonal antibody.
This approval was based on data from the phase 2 Horizon clinical trial evaluating efficacy of the doublet in patients with relapsed or refractory MM (n = 97). Study participants were given melphalan flufenamide 40 mg on day 1 plus dexamethasone 40 mg on days 1, 8, 15, and 22. Of note, patients aged 75 years or older were given 20 mg of dexamethasone instead of 40 mg.
Treatment was administered in 28-day cycles until disease progression or unacceptable toxicity occurred. Study findings showed that the overall response rate was 23.7% (95% CI, 15.7-33.4) and the median duration of response was 4.2 months (95% CI, 2.3-7.6).
Among 157 patients included in the safety portion of the study, the most common (>20%) adverse reactions associated with the combination therapy included fatigue, nausea, diarrhea, pyrexia, and respiratory tract infection. The most common (≥50%) laboratory abnormalities were decreased leukocytes, platelets, lymphocytes, neutrophils, and hemoglobin, and increased creatinine.
Of note, the FDA has said that the safety and efficacy of melphalan flufenamide has not been established for use as a conditioning regimen in patients undergoing transplantation.—Emily Bader
Source: US Food and Drug Administration. FDA approves FDA grants accelerated approval to melphalan flufenamide for relapsed or refractory multiple myeloma. February 26, 2021. www.fda.gov/drugs/drug-approvals-and-databases/fda-grants-accelerated-approval-melphalan-flufenamide-relapsed-or-refractory-multiple-myeloma. Accessed March 3, 2021.
