Leukemia Treatment Approved by FDA

The FDA today approved venetoclax (Venclexta) for the treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion and who have been treated with a least 1 prior therapy.

This is the first FDA approved treatment that inhibits the B-cell lymphoma 2 (BCL-2) protein.

“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 at 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 approval was based on a single-arm clinical trial that enrolled 106 patients with CLL who have a 17p deletion and had at least 1 prior therapy. Participants were administered a 20-mg oral dose of Venclexta that increased over a 5-week period to 400-mg.

The results of the study showed that 80% of patients experienced a complete or partial remission of their cancer. The most common side effects included neutropenia, anemia, nausea, diarrhea, upper respiratory tract infection, thrombocytopenia, and fatigue.

Serious complications included pneumonia, neutropenia with fever, fever, autoimmune hemolytic anemia, and tumor lysis syndrome.

“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.”