New leukemia drug available

April 13, 2016

FDA recently approved venetoclax (Venclexta, AbbVie and Genentech) to treat chronic lymphocytic leukemia (CLL) who have a chromosomal abnormality called 17p deletion and who have been treated with at least 1 prior therapy

FDA recently approved venetoclax (Venclexta, AbbVie and Genentech) to treat chronic lymphocytic leukemia (CLL) for patients who have a chromosomal abnormality called 17p deletion and who have been treated with at least 1 prior therapy.

“Up to half of people whose CLL progressed have 17p deletion, a genetic marker that makes the disease difficult-to-treat,” said Sandra Horning, MD, chief medical officer and head of global product development for Genentech. “Venclexta is the first approved medicine designed to trigger a natural process that helps cells self-destruct, and is a new way to help people who have been previously treated and have this high-risk form of the disease.”

The medication should be available in the United States next week and costs around $109,500 for the first year. However, "Genentech is committed to ensuring its medicines get to the people who need them, even if they can’t afford them," a Genentech spokesperson told FormularyWatch.

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According to the National Cancer Institute, CLL is one of the most common types of leukemia in adults, with approximately 15,000 new cases diagnosed each year. CLL is characterized by the progressive accumulation of abnormal lymphocytes, a type of white blood cell.

Patients with CLL who have a 17p deletion lack a portion of the chromosome that acts to suppress cancer growth. This occurs in approximately 10% of patients with untreated CLL and in approximately 20% of patients with relapsed CLL.

“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 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.”

Related: FDA expands approved use of Imbruvica for rare form of non-Hodgkin lymphoma

The efficacy of Venclexta was tested in a single-arm clinical trial of 106 patients with CLL who have a 17p deletion and who had received at least 1 prior therapy. Trial participants took Venclexta orally every day, beginning with 20 mg. and increasing over a 5-week period to 400 mg. Results showed that 80% of trial participants experienced a complete or partial remission of their cancer. 

Venclexta is indicated for daily use after detection of 17p deletion is confirmed through the use of the FDA-approved companion diagnostic Vysis CLL FISH probe kit.

The most common side effects of Venclexta include low white blood cell count (neutropenia), diarrhea, nausea, anemia, upper respiratory tract infection, low platelet count (thrombocytopenia) and fatigue. Serious complications can include pneumonia, neutropenia with fever, fever, autoimmune hemolytic anemia, anemia and metabolic abnormalities known as tumor lysis syndrome. Live attenuated vaccines should not be given to patients taking Venclexta.

Read more: FDA approves Gazyva sBLA with new data in previously untreated CLL