FDA approves drug for rare type of leukemia

The agency increased the speed of approval after 80 percent of patients in a clinical trial responded to the drug.

By Stephen Feller

BETHESDA, Md., April 11 (UPI) -- The U.S. Food and Drug Administration on Monday approved a new drug for patients with a rare form of leukemia who were not helped by other treatments.

Venclexta, manufactured by AbbVie, was given priority review status and accelerated approval by the FDA after more than three-quarters of patients responded to the drug in clinical trials.


The drug is meant for chronic lymphocytic leukemia patients who have a genetic mutation called 17p deletion and have no response to at least one other form of treatment, of which there are about 15,000 new cases per year. The abnormality is found in about 10 percent of patients just diagnosed with CLL and 20 percent of people with relapsed CLL.

The benefit of Venclexta is that the drug inhibits the BCL2 protein, which supports cancer growth in CLL patients who have the 17p deletion, which helps to avoid killing other cells and making health worse the way many cancer treatments do, Michael Severino, AbbVie's chief scientific officer, told Business Insider.

In a single-arm trial, researchers recruited 106 patents to receive Venclexta each day, starting with a 20 milligrams per day and increasing over five weeks to 400 milligrams per day, and 80 percent of patients responded to the drug.


Venclexta was also approved by the FDA in January to be used with rituximab for relapsed and refractory chronic lymphocytic leukemia, as well as for acute myeloid leukemia.

"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."

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