FDA In Brief: FDA warns about safety concerns related to investigational use of Venclexta (venetoclax) for multiple myeloma

March 21, 2019

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"Clinical trials are crucial to advancing new therapies to treat patients with life-threatening disease, like cancer. Every day, thousands of trials investigate experimental therapies and new uses for existing therapies. While much effort is put into early phase research to help ensure that no major safety issues exist before therapies are tested in people, these trials are ultimately designed to provide vital information about safety and effectiveness, so the FDA can help ensure that the benefits to the intended patients outweigh the risks. When significant safety issues do arise, we work quickly to manage these issues. Today the FDA is announcing the suspension of several clinical trials using Venclexta in combination with other drugs to treat patients with multiple myeloma because of an increased risk of death compared to the control group. This announcement does not affect patients who are taking Venclexta for approved uses. The FDA is actively examining the data from the trial and working directly with the drug’s sponsor to better understand the cause of the safety concerns. The FDA will take any additional actions as warranted to help ensure patients enrolled in this trial are protected and that doctors and clinical trial researchers understand the risks associated with this investigational use,” said FDA’s Center for Drug Evaluation and Research director Janet Woodcock, M.D. “Today’s alert underscores the importance of why new therapies are thoroughly studied to help ensure their benefits outweigh the risks to patients, and we will continue to carefully monitor clinical trials to help ensure patients are protected when safety concerns arise."

Today, the FDA posted a safety statement to alert health care professionals and patients about the suspension of a clinical trial of bortezomib and low-dose dexamethasone with or without Venclexta in patients with relapsed and refractory multiple myeloma, due to an increased risk of death for patients receiving Venclexta when it was combined with bortezomib and dexamethasone as compared to the control group. The FDA has required that patients in this clinical trial be discontinued from further treatment, because interim results from the trial demonstrate an increased risk of death for patients receiving Venclexta when it was combined with bortezomib as compared to the control group. No new patients will be enrolled and only patients who are deriving clinical benefit will continue treatment in this clinical trial after reconsent. Other multiple myeloma clinical trials of Venclexta and other combinations were also suspended.

The FDA is communicating now, given the serious nature of the safety issue, to remind doctors and patients that Venclexta is not FDA-approved to treat multiple myeloma. The FDA’s safety alert does not apply to the approved indications for Venclexta as the safety and efficacy of using Venclexta for approved, on-label uses have been established.

Patients with questions about their treatment with Venclexta should contact their health care provider. Health care professionals and patients are encouraged to report any adverse events or side effects related to the use of any drugs to the FDA’s MedWatch Adverse Event Reporting program.

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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.