FDA Issues Statement on Keytruda Clinical Trial Holds

Pembrolizumab (Keytruda) combination therapy may increase mortality risk among multiple myeloma patients.

Based on data from 2 clinical trials of pembrolizumab (Keytruda), the FDA has issued a statement to inform the general public, healthcare providers, and clinical investigators about the risks associated with pembrolizumab plus dexamethasone and lenalidomide or pomalidomide for patients with multiple myeloma, according to a press release.

The FDA reviewed data from the KEYNOTE-183 and KEYNOTE-185 trials, which evaluated pembrolizumab as part of a combination therapy for multiple myeloma.

Previously, the FDA halted the clinical trials due to evidence that suggests pembrolizumab plus an immunomodulatory agent can result in an increased risk of death compared with the control group, according to the release.

Merck was informed of the potential risk of death and stopped clinical testing on July 12, 2017.

“The FDA will take appropriate action as warranted to ensure patients enrolled in these trials are protected and that doctors and clinical trial researchers understand the risks associated with this investigational use,” Janet Woodcock, director of the FDA Center for Drug Evaluation and Research, said in a statement.

Currently, pembrolizumab is approved to treat melanoma, lung cancer, head and neck cancer, classical Hodgkin lymphoma, urothelial carcinoma, and microsatellite instability-high cancer. Pembrolizumab is currently not approved to treat multiple myeloma.

The FDA advises that the potential increased mortality risk does not apply to patients taking pembrolizumab for an approved indication and that those patients should continue treatment as directed, according to the release.

“We also want to ensure that patients taking Keytruda and other PD-1/PD-L1 inhibitors know that the FDA still believes the benefits of taking these drugs for their approved uses and as indicated in the labels continue to outweigh their risks,” Dr Woodcock said. “Patients taking these drugs for their approved uses should continue to take their medication as directed by their healthcare professional.”

Other clinical trials of pembrolizumab, PD-1/PD-L1 cancer drugs, and other combination therapies for multiple myeloma are currently undergoing investigation, according to the FDA.

The FDA will work with Merck, sponsors of other PD-1/PD-L1 cancer drugs, and clinical investigators to determine the impact of the potential issue and will alert the public as necessary.

“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,” Dr Woodcock concluded.