FDA Warns Patients With Myeloma About Pepaxto: Drug Increased Death Rates in Clinical Trial

The Food and Drug Administration (FDA) recently alerted patients and clinicians about Pepaxto (melphalan flufenamide) plus dexamethasone for the treatment of patients with multiple myeloma. According to findings from the OCEAN, Study OP-103 trial, the drug regimen led to an increased risk of death.

In February of this year, the FDA granted Pepaxto plus dexamethasone an accelerated approval to treat patients with relapsed/refractory myeloma who have received four or more prior lines of therapy and whose disease was refractory (unresponsive) to at least one proteasome inhibitor, one immunomodulatory agent and one CD38-directed monoclonal antibody.

Drugs granted accelerated approval are required to be tested in more clinical trials to ensure that they are safe and effective. In this case, the OCEAN trial showed that 48% of patients in the Pepaxto group died, compared to 43% of patients who were given Pomalyst (pomalidomide). Average overall survival was also shorter for patients in the Pepaxto group at 19.7 months, compared to 25 months in the Pomalyst group, though the difference may not be statistically significant.

Since there was no survival benefit and an increased risk of death with Pepaxto, the FDA required Oncopeptides AB, the manufacturer of Pepaxto, to stop enrollment in OCEAN and any other ongoing trials involving the drug. If patients on the OCEAN trail are receiving clinical benefit from Pepaxto, they can choose to continue treatment with it if they are informed of the risks and sign a revised written informed consent.

Patients with myeloma receiving Pepaxto treatment outside of a clinical trial should discuss the potential risks and benefits with their health care team.

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