FDA revokes approval of cancer drug Pepaxto

More than two and a half years after the FDA warned about an increased risk of death linked to Pepaxto, a multiple myeloma drug, the agency withdrew the medication’s approval Feb. 23. 

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Pepaxto (melphalan flufenamide) with dexamethasone gained accelerated approval in February 2021, but the therapy failed a confirmatory trial. The manufacturer, Oncopeptides, then suspended enrollment. In July 2021, the FDA said there was a “detrimental effect on overall survival” in the study. 

About a year later, an agency committee voted 14-2 in concluding an unsafe benefit-risk ratio. In August 2023, the FDA proposed pulling the approval, and the decision was finalized Feb. 23. 

In a news release, the agency said “it is FDA’s understanding that Oncopeptides does not currently market Pepaxto in the U.S.”

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