Breakthrough therapy designation for venetoclax for treatment of patients with untreated (treatment-naïve) acute myeloid leukemia (AML)-AbbVie

AbbVie has announced that the 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 being developed by AbbVie in partnership with Genentech and Roche.

AML is characterized by the body producing too many of a specific type of white blood cell (myeloblast) which can crowd out healthy blood cells. In the U.S. specifically, it was estimated that more than 20,000 people would be diagnosed with AML in 2015. AML 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 AML and reported in one investigational study. AML 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 AML, 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 percent, these patients are best managed with non-intensive approaches. HMAs are currently used as monotherapy for treatment of AML 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. HMAs, alone or in combination with the investigational agent venetoclax is not approved for the treatment of AML.