The FDA has accepted the New Drug Application (NDA) and granted Priority Review for Roche Group member Genentech’s venetoclax for the treatment of people with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy, including those with 17p deletion. Venetoclax is a small molecule inhibitor of the BCL-2 protein being developed in partnership with AbbVie, and was granted Breakthrough Therapy designation by the FDA in April 2015 for the treatment of people with previously treated (relapsed or refractory) CLL with 17p deletion.

“Venetoclax is a potential new way to treat this difficult type of chronic lymphocytic leukemia,” said Sandra Horning, M.D., chief medical officer and head of global product development. “We look forward to working with AbbVie and health authorities to bring this first-of-its-kind medicine to people who need more options.”

A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. Breakthrough Therapy designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible. A Marketing Authorization Application (MAA) also has been validated by the EMA.

The NDA for venetoclax is based in part on data from the pivotal phase II M13-982 study. M13-982 (NCT01889186) is a phase II, open-label, single arm, multicenter study evaluating the efficacy and safety of venetoclax in patients with relapsed, refractory or previously untreated CLL with 17p deletion. The study included 107 patients with relapsed or refractory disease, and all but one had 17p deletion. Additionally, about 50 patients with relapsed, refractory or previously untreated disease have been enrolled in the safety expansion cohort. The primary endpoint of the study is overall response rate as determined by an independent review committee, and secondary endpoints include complete response, partial response, duration of response, progression-free survival and overall survival. The level of minimal residual disease in peripheral blood and/or bone marrow was assessed in a subset of patients.

CLL is one of the most common forms of blood cancer, and each year there are an estimated 5,000 deaths from CLL in the U.S. Although signs of CLL may disappear for a period of time after initial treatment, the disease is considered incurable and many people will require additional treatment due to the return of cancerous cells.