The U.S. Food and Drug Administration (FDA) has approved venetoclax (Venclexta) for the treatment of patients with chronic lymphocytic leukemia (CLL) and a confirmed 17p deletion with the FDA-approved companion diagnostic Vysis CLL FISH probe kit who have received at least 1 prior therapy.1
Accelerated approval was based on findings from a single-arm clinical trial that evaluated the safety and efficacy of venetoclax in 106 patients with CLL who have a 17p deletion and who were treated with at least 1 prior therapy.
Participants received venetoclax orally daily starting at 20 mg and increasing to 400 mg over 5 weeks. Results showed that 80% of patients achieved a complete or partial remission.
The most frequently reported adverse events were neutropenia, diarrhea, nausea, anemia, upper respiratory tract infection, thrombocytopenia, and fatigue.
Clinicians should be aware that venetoclax may cause serious complications, including pneumonia, pyrexia, neutropenic fever, autoimmune hemolytic anemia, anemia, and tumor lysis syndrome.
Patients should not receive live attenuated vaccines, such as the MMR (measles, mumps, rubella) vaccine, the varicella vaccine, and the seasonal flu nasal spray vaccine, while receiving venetoclax.
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“These patients now have a new, targeted therapy that inhibits a protein involved in keeping tumor cells alive,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “For certain patients with CLL who have not had favorable outcomes with other therapies, Venclexta may provide a new option for their specific condition.”
Venetoclax is a small molecule Bcl-2 inhibitor that blocks anti-apoptotic B-cell lymphoma (Bcl-2) protein, thereby causing CLL cell death.
- FDA approves new drug for chronic lymphocytic leukemia in patients with a specific chromosomal abnormality [news release]. Silver Spring, MD: U.S. Food and Drug Administration; April 11, 2016. http://www.fda.gov/NewsEvents/Newsroom/ PressAnnouncements/ucm495253.htm. April 11, 2016.