The U.S. Food and Drug Administration has granted a new breakthrough therapy designation to venetoclax for use in combination with hypomethylating agents (HMAs) for the treatment of patients with untreated acute myeloid leukemia who are ineligible to receive standard induction therapy, high-dose chemotherapy. Venetoclax is an inhibitor of the B-cell lymphoma-2 (BCL-2) protein.
The breakthrough therapy designation, enacted as part of the FDA's 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.
Venetoclax has previously received breakthrough therapy designation for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) with the 17p deletion genetic mutation (OT 6/25/15 issue); and earlier this year the drug received another breakthrough therapy designation for the treatment of patients with relapsed or refractory CLL with the 17p deletion mutation for use in combination with rituximab (OT 2/25/15 issue).
This breakthrough therapy designation for venetoclax is based on data from a Phase II clinical trial that were presented at the American Society of Hematology Annual Meeting in 2014.
Venetoclax is being developed in collaboration with Genentech and Roche.