Marking another promising advance for the treatment of patients with chronic lymphocytic leukemia, including those with a rare subset of this blood cancer, the U.S. Food and Drug Administration (FDA) has granted priority review for an investigational compound that has shown positive results in a Phase II clinical trial.
Venetoclax has shown great potential as a new way of treating CLL patients who have received at least one prior therapy. It also appears to be effective for patients with a rare subset in which a piece of chromosome 17 is missing. Venetoclax works by inhibiting the BCL-2 protein and enabling a signaling system that tells cells, including cancer cells, to self-destruct.
More than 126,000 patients in the U.S. currently live with CLL, a typically slow-moving blood cancer that begins in the bone marrow. Of those CLL patients who do not respond to therapy, or who have relapsed, approximately 30 percent are found to have a mutation in which they are missing part of chromosome 17.
A Priority Review designation is granted to medicines that the FDA believes have the potential to provide significant improvement in the treatment, prevention or diagnosis of a disease. The compound was granted a Breakthrough Therapy Designation in April 2015 in order to expedite its development and review.