Breakthrough Therapy Designation for Venetoclax in AML

Venetoclax (Venclexta), in combination with low-dose cytarabine, has been granted a breakthrough therapy designation for use in elderly, treatment-naive patients with acute myeloid leukemia (AML) who cannot handle intensive chemotherapy.

A small-molecule inhibitor of BCL-2, venetoclax promotes cancer cell apoptosis or programmed cell death. The current designation was granted following an FDA review of a phase 1b open-label study of venetoclax with cytarabine in individuals aged 65 or older diagnosed with AML. The drug was shown to have durable efficacy and an acceptable safety profile in combination with low-dose cytarabine in this patient population.

According to a study presented at the annual meeting of the American Society of Hematology in 2016, clinical remission was achieved in a majority of the 20 patients enrolled in the study, and median overall survival was not documented at the time of the presentation. A substantially better survival in responders, as compared to nonresponders, suggests that the improvement is likely due to treatment with venetoclax plus low-dose cytarabine, the authors noted, which included:

• An objective response rate in 15 patients (75%)
• A complete response in 14 patients (70%)
• Bone marrow blast percentage below 5% in 16 of 19 patients (84%)

Venetoclax was previously approved as second-line treatment for chronic lymphocytic leukemia in patients who harbor a 17p deletion. It is also an “exciting drug” for the treatment of non-Hodgkin’s lymphoma, according to Craig Portell, MD, University of Virginia Health System.

Developed by AbbVie and Roche, the treatment will be commercialized by AbbVie and Genentech.