FDA Warns of Risk of Using Cancer Drug for Unapproved Indication

By Jonathan Block

The FDA is reminding both patients and doctors that the cancer drug Keytruda (pembrolizumab) is not approved for use in multiple myeloma after an unusual number of deaths in clinical trials investigating Keytruda in combination with 2 other therapies for the blood cancer.

Two trials examining Keytruda in combination with immunomodulators such as Revlimid (lenalidomide) and Pomalyst (pomalidomide) for multiple myeloma were put on hold in July after investigators reported an “excess” number of deaths in those being treated with Keytruda compared to those in a control arm that did not include the drug. At that time, researchers stopped giving Keytruda to patients in the trials.

“Today’s alert underscores the importance of why new therapies are thoroughly studied to ensure the benefits of taking them outweigh the risks to patients, and we will continue to aggressively monitor clinical trials to ensure patients are protected when safety concerns arise,” Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said in a statement.

The FDA also said that if patients are taking Keytruda for approved uses — which include head and neck cancer, Hodgkin lymphoma and melanoma — they should continue taking the medication, as the agency believes the benefits outweigh the risks for these conditions.