Pepaxto Associated with Increased Risk of Death in a Clinical Trial

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Pepaxto Associated with Increased Risk of Death in a Clinical Trial

July 28, 2021 – Pepaxto® (melphalan flufenamide – Oncopeptides AB) may increase the risk of death for patients who have multiple myeloma, according to the U.S. FDA. In 2021, the agency granted accelerated approval for Pepaxto to be used in combination with dexamethasone for treatment of certain adults diagnosed with relapsed or refractory multiple myeloma.

As a condition of the accelerated approval, Pepaxto’s manufacturer began the OCEAN trial, comparing treatment with Pepaxto and dexamethasone to treatment with pomalidomide and dexamethasone. The FDA is now calling to suspend enrollment in the trial and other ongoing Pepaxto clinical trials. A statistical analysis of OCEAN’s results found a higher rate of mortality in the Pepaxto and dexamethasone arm (48%) compared to the pomalidomide and dexamethasone arm (43%). Median overall survival was also shorter with Pepaxto and dexamethasone (19.7 months) versus pomalidomide and dexamethasone (25 months).

The FDA recommends healthcare professionals speak with patients who are taking Pepaxto regarding risks versus benefits of staying on the medication. The agency has also stated that patients receiving clinical benefit from the drug in the OCEAN trial may continue to take it if they are advised of the risks and give informed consent.

Pepaxto has no other FDA-approved indications, and the agency has indicated it may hold a public meeting to discuss continued marketing of the drug.