The Food and Drug Administration (FDA) issued a safety alert for Pepaxto (melphalan flufenamide; Oncopeptides AB) based on results from the OCEAN trial, which demonstrated an increased risk of death.

In February 2021, the FDA approved Pepaxto under Accelerated Approval for use in combination with dexamethasone to treat adult patients with relapsed or refractory multiple myeloma who have received at least 4 prior lines of therapy and whose disease was refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent, and 1 CD38-directed monoclonal antibody. The manufacturer was required to conduct the OCEAN trial as a post-approval requirement under the accelerated approval program.

The trial compared Pepaxto plus low-dose dexamethasone with pomalidomide plus low-dose dexamethasone in patients with relapsed or refractory multiple myeloma following 2-4 lines of prior therapy and in patients who were resistant to lenalidomide in the last line of therapy. 

Due to the detrimental effect on overall survival in the OCEAN trial, the FDA is requiring the manufacturer suspend enrollment in the trial. The FDA has also suspended enrollment in other ongoing Pepaxto clinical trials. Patients receiving clinical benefit from Pepaxto may continue treatment in the OCEAN trial provided they are informed of the risks and sign a revised written informed consent.

The FDA continues to evaluate the OCEAN trial results and may hold a future public meeting to discuss these safety findings and explore the continued marketing of Pepaxto. The agency will update patients and health care professionals when new information is available.

Food and Drug Administration (FDA). FDA alerts patients and health care professionals about clinical trial results showing an increased risk of death associated with Pepaxto (melphalan flufenamide) [news release]. July 28, 2021. Available at: click here. Accessed July 28, 2021.