FDA grants third venetoclax Breakthrough Therapy Designation

28 January 2016  •  Author: Victoria White

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for venetoclax in combination with hypomethylating agents (HMAs) for the treatment of patients with untreated (treatment-naïve) acute myeloid leukaemia (AML) who are ineligible to receive standard induction therapy.


Venetoclax is an investigational oral B-cell lymphoma-2 (BCL-2) inhibitor being evaluated for the treatment of patients with various cancer types. The BCL-2 protein prevents apoptosis (programmed cell death) of some cells, including lymphocytes, and can be over expressed in some cancer types. Venetoclax is designed to selectively inhibit the function of the BCL-2 protein. The therapy is being developed by AbbVie in collaboration with Genentech and Roche.

The Breakthrough Therapy Designation is supported by data from untreated patients, 65 years or older with AML and reported in one investigational study.

Third Breakthrough Therapy Designation for venetoclax

Commenting on the announcement, Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie, said: “Acute myeloid leukaemia is an aggressive and life- threatening cancer. Unfortunately, for many AML patients, intensive therapy is not an option, causing a high need for new, effective, and alternative treatments. This third Breakthrough Therapy Designation for venetoclax highlights our efforts to pursue the significant and broad potential of this therapy.”

In April 2015, the FDA granted Breakthrough Therapy Designation to single agent venetoclax for the treatment of CLL in previously treated (relapsed/refractory) patients with the 17p deletion genetic mutation. In January 2016, AbbVie announced that the FDA granted priority review for the single agent NDA application, and granted a second Breakthrough Therapy Designation for venetoclax supported by the investigational study in combination with rituximab for the treatment of patients with relapsed/refractory chronic lymphocytic leukaemia (R/R CLL). According to the FDA, Breakthrough Therapy Designation is intended to expedite the development and review of therapies for serious or life-threatening conditions.

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