The FDA has issued an alert regarding melphalan flufenamide (Pepaxto)

July 29, 2021

On July 28, 2021, the FDA announced that the phase 3, OCEAN clinical trial (NCT03151811) showed an increased risk of death associated with melphalan flufenamide (Pepaxto) with dexamethasone for the treatment of the patients with multiple myeloma.

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Last February, the FDA granted an accelerated approval to melphalan flufenamide (Pepaxto; melflufen) with dexamethasone for the multiple myeloma patients whose cancer progressed even after multiple lines of treatment. The approval was based on the result of the phase 2 HORIZON study (NCT02963493), 23.7 % of patients responded to the drugs for median 4.2 months (please see our post in March 1, 2020). 

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Unfortunately, the results from the phase 3 OCEAN study (NCT03151811) showed that the patient received melphalan flufenamide/dexamethasone survived for 19.7 months overall, compared to those treated with pomalidomide/dexamethasone for 25 months. Based on this finding, the FDA concluded that melphalan flufenamide/dexamethasone has a detrimental effect on the survival of patients.

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The FDA ordered the manufacturer to suspend enrollment in the OCEAN trial as well as other ongoing clinical trials with melphalan flufenamide. However, the FDA says that the patients receiving clinical benefit from melphalan fluefenamide may continue treatment in the OCEAN trial only when they are informed of the risks and sign a revised written informed consent.