FDA Approves AbbVie’s Drug For Rare Leukemia

The US Food and Drug Administration (FDA) has approved AbbVie’s Venclexta as a second-line treatment for chronic lymphocytic leukemia, in patients with a chromosomal abnormality known as a 17p deletion. The drug is the first FDA-approved treatment that targets a key protein – known as B-cell lymphoma 2 (BCL-2) protein – involved in cancer cell growth.

Chronic lymphocytic leukemia is among the most common types of leukemia in adults, according to the National Cancer Institute. The disease is characterized by the accumulation of abnormal lymphocytes, and 15,000 new cases of the cancer are diagnosed each year.

Patients with chronic lymphocytic leukemia who also have a deletion on the short arm of the 17th chromosome – known as a 17p mutation – lack the genes necessary to suppress the growth of cancer cells. Around 10 percent of patients with untreated forms of chronic lymphocytic leukemia have this mutation, while closer to 20 percent of patients with relapsed leukemia have the chromosomal abnormality.

“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 chronic lymphocytic leukemia who have not had favorable outcomes with other therapies, Venclexta may provide a new option for their specific condition.”

Venclexta was tested in a single-arm clinical trial of leukemia patients with the 17p mutation, who had previously been treated with at least one other therapy. Patients were given an increasing daily dose of the drug, starting with 20mg and reaching a peak dose of 400mg at five weeks of treatment.

The results of the trial showed that 80 percent of patients experienced full or partial remission of their cancer after receiving Venclexta. “BCL-2 inhibition is a novel mechanism which brings a new treatment option to patients who need additional therapies,” said AbbVie CEO, Richard Gonzalez.

Venclexta received a number of advantageous designations from the FDA, including priority review status, accelerated approval, breakthrough therapy and orphan drug status. AbbVie and Roche’s Genentech will both be responsible for marketing the drug in the US.