FDA approves Venclexta for previously treated CLL with 17p deletion

The FDA today approved venetoclax for the treatment of patients with chronic lymphocytic leukemia who have deletion of chromosome 17p and who have received at least one prior therapy.

Venetoclax (Venclexta; AbbVie, Genentech) is the first FDA–approved therapy that targets the BCL-2 protein, which supports cancer cell growth and often is overexpressed in CLL.

“This FDA approval marks a major milestone for our company and, more importantly, for the patients diagnosed with relapsed/refractory CLL who harbor the 17p deletion,” Richard Gonzalez, chairman of the board and CEO of AbbVie, said in a statement. “BCL-2 inhibition is a novel mechanism which brings a new treatment option to patients who need additional therapies.”

Approximately 10% of patients with CLL and 20% of patients with relapsed CLL have deletion of 17p, a part of the chromosome that suppresses cancer growth. Venetoclax is intended for use in patients after 17p deletion is confirmed with the FDA–approved companion diagnostic Vysis CLL FISH probe kit (Abbott Molecular).

Sandra Horning

“Up to half of people whose CLL progressed have 17p deletion, a genetic marker that makes the disease difficult-to-treat,” Sandra Horning, MD, chief medical officer and head of global product development at Genentech, said in a statement. “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 FDA based its decision in part on the results of a single-arm trial of 106 patients with CLL and 17p deletion who had received at least one prior therapy. Patients received doses of venetoclax that increased from 20 mg to 400 mg over a 5-week period.

Overall, 80% of the patients achieved a complete or partial remission.

The most common adverse events associated with venetoclax include neutropenia, diarrhea, nausea, anemia, upper respiratory tract infection, thrombocytopenia and fatigue. Serious adverse events can include pneumonia, neutropenia with fever, fever, autoimmune hemolytic anemia, anemia and tumor lysis syndrome.

Richard Pazdur

Venetoclax previously received breakthrough therapy designation, priority review status, accelerated approval and orphan drug designation for this indication.

“These patients now have a new, targeted therapy that inhibits a protein involved in keeping tumor cells alive,” Richard Pazdur, director of the office of hematology and oncology products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “For certain patients with CLL who have not had favorable outcomes with other therapies, Venclexta may provide a new option for their specific condition.”