The Food and Drug Administration ( FDA ) has granted Breakthrough Therapy designation for the investigational agent Venetoclax in combination with hypomethylating agents ( HMAs ) for the treatment of patients with untreated ( treatment-naïve ) acute myeloid leukemia ( AML ) who are ineligible to receive standard induction therapy ( high-dose chemotherapy ).
Venetoclax is an inhibitor of the B-cell lymphoma-2 ( BCL-2 ) protein.

Acute myeloid leukemia is the most common type of acute leukemia among adults. The average age of a patient with AML is about 67 years.

The Breakthrough Therapy designation is supported by data from untreated patients, 65 years or older with acute myeloid leukemia and reported in one investigational study.
Acute myeloid leukemia is typically treated with chemotherapy and the intensity of chemotherapy depends on a person's age and health. However, no single standard of care exists for older patients with acute myeloid leukemia, as there are no FDA-approved therapies for patients not eligible to receive standard induction therapy / intensive chemotherapy.
Because induction-therapy-related mortality for patients 55 years of age or older has been estimated at 15 to 20%, these patients are best managed with non-intensive approaches.
Hypomethylating agents are currently used as monotherapy for treatment of acute myeloid leukemia in the United States, as low-intensity therapy which may be appropriate for elderly patients, or those with comorbidities who cannot tolerate standard induction therapy.

In April 2015, the FDA granted Breakthrough Therapy designation to single agent Venetoclax for the treatment of chronic lymphocytic leukemia ( CLL ) in previously treated ( relapsed / refractory ) patients with the 17p deletion genetic mutation.
In January 2016, 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 leukemia ( R/R CLL ).

Venetoclax is an investigational oral BCL-2 inhibitor. 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. ( Xagena )

Source: Abbvie, 2016