AbbVie , a global biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) 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.
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.
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 leukemia (R/R CLL).
