Melphalan flufenamide receives approval from the FDA for relapsed or refractory multiple myeloma

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August 2021: Melphalan flufenamide (Pepaxto, Oncopeptides AB) in combination with dexamethasone has been granted accelerated approval by the Food and Drug Administration for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody.

HORIZON (NCT02963493), a multicenter, single-arm trial, was used to assess efficacy. Patients had to have relapsed refractory multiple myeloma to be eligible. Patients received 40 mg of melphalan flufenamide intravenously on day 1 and 40 mg of dexamethasone orally on days 1, 8, 15, and 22 of each 28-day cycle until disease progression or intolerable toxicity.

Efficacy was assessed in a subset of 97 patients who had failed at least one proteasome inhibitor, one immunomodulatory drug, and a CD38-directed antibody and had had four or more prior lines of therapy. The overall response rate (ORR) and duration of response (DOR) were the primary efficacy outcomes measured by investigators using the International Myeloma Working Group (IMWG) Criteria. The ORR was 23.7 percent (95 percent confidence interval: 15.7 to 33.4), with a median DOR of 4.2 months (95 percent CI: 3.2, 7.6).

The 157 participants enrolled in HORIZON were examined for safety. Fatigue, nausea, diarrhoea, pyrexia, and respiratory tract infection are the most common side effects (> 20%). Decreased leukocytes, platelets, lymphocytes, neutrophils, and haemoglobin, as well as increased creatinine, are the most prevalent laboratory abnormalities (50 percent).

Melphalan flufenamide’s safety and efficacy as a conditioning regimen in transplant recipients has yet to be determined. Outside of controlled clinical trials, melphalan flufenamide is not indicated and is not recommended for use as a conditioning regimen for transplant, according to the USPI.

The recommended dose of melphalan flufenamide in conjunction with dexamethasone is 40 mg intravenously over 30 minutes on day 1 of each 28-day therapy cycle.


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