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FDA Statement

Statement from Center for Drug Evaluation and Research Director Janet Woodcock regarding safety concerns related to investigational use of Keytruda in multiple myeloma

For Immediate Release:
Statement From:

Clinical trials play a critically important role in bringing to market innovative new therapies for patients facing life-threatening disease. It is an exciting time for medical innovation. Already, clinical trials have helped realize exciting and meaningful advancements for patients with critical diseases, like cancer, and thousands of clinical trials are currently underway looking at experimental therapies, combinations of therapies and new uses for existing therapies.

But with this hope comes the understanding that there are inherent risks for patients who are willing to participate in this type of research. While much effort is put into early research to ensure that no major safety issues exist before therapies are tested in patients, these trials are ultimately designed to provide vital information about the safety and efficacy of the treatments in people so the FDA can help ensure that the benefits to the intended patients outweigh the risks. When significant safety issues do arise, we must work quickly to prevent further injury or deaths.

This was recently the case in two clinical trials for an investigational use of the cancer drug Keytruda (pembrolizumab) in combination with two other therapies. Scientists monitoring the studies found an excess of deaths for patients receiving Keytruda when combined with these other drugs.

As is required in these scenarios, once the safety issue was discovered, trial sponsor Merck and the FDA took immediate action to protect patient safety. Merck stopped enrolling patients into these trials and reported initial concerns to the agency in June 2017. After receiving more information provided by Merck, the FDA acted swiftly in placing a full clinical hold on these trials last month. Patients are no longer receiving treatment with Keytruda in these two trials, which were studying Keytruda for a use that has not been evaluated or approved by the agency.

The FDA is actively examining the data from the Keytruda trials and working directly with Merck to better understand the true cause of the safety concerns. In addition, the agency is working with sponsors of other similar cancer drugs, known as PD-1/PD-L1 inhibitors, to examine other trials in which these drugs are being studied in combination with other drugs, known as immunomodulatory agents, and in which they’re being studied in combination with other classes of drugs in hematologic malignancies. 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.

We are communicating now, given the serious nature of the safety issue, to remind doctors and patients that Keytruda is not approved for the treatment of multiple myeloma and should not be given to patients in combination with any immunomodulatory agents, including Revlimid (lenalidomide) and Pomalyst (pomalidomide), for the treatment of multiple myeloma.

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. Patients taking these drugs for their approved uses should continue to take their medication as directed by their health care professional. 

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.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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Angela Stark

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