ISSUE: FDA is alerting patients and health care professionals that a clinical trial (OCEAN, Study OP-103) evaluating Pepaxto (melphalan flufenamide) with dexamethasone to treat patients with multiple myeloma showed an increased risk of death.  

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

BACKGROUND: In February 2021, FDA approved Pepaxto for use in combination with dexamethasone to treat adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease was refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.

RECOMMENDATIONS:  

• FDA encourages health care professionals to review patients’ progress on Pepaxto and discuss the risks of continued administration with each patient in the context of other treatments. 
• Patients currently receiving Pepaxto should also discuss with their health care professional the risks and benefits of receiving Pepaxto.

FDA encourages health care professionals and patients to report adverse events or quality problems experienced with the use of any medication to FDA’s MedWatch Adverse Event Reporting program: 

• Complete and submit the report online; or
• Download and complete the form, then submit it via fax at 1-800-FDA-0178