Venetoclax Granted Breakthrough Designation for Acute Myeloid Leukemia

Venetoclax is an inhibitor of the B-cell lymphoma-2 protein used in combination with hypomethylating agents in treatment-naive AML patients.

The FDA had granted Breakthrough Therapy Designation (BTD) to a new treatment option for acute myeloid leukemia (AML).

AbbVie’s venetoclax is an inhibitor of the B-cell lymphoma-2 (BCL-2) protein. The agent is used in combination with hypomethylating agents (HMA) in treatment-naïve AML patients who are ineligible to receive high-dose chemotherapy.

With AML, the body produces too many myeloblasts — an immature white blood cell – that crowds out other healthy blood cells. AML is the most common type of acute leukemia and in the United States. Approximately 20,000 people were diagnosed with the disease in 2015.

"Acute myeloid leukemia is an aggressive and life-threatening cancer,” said Executive Vice President of AbbVie, Michael Severino, MD. “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."

The BTD followed an investigational study that showed data from patients aged 65 years or older with AML that was left untreated. Although most patients are treated with chemotherapy, the intensity of chemotherapy depends on how old a person is and what condition their health is in.

Unfortunately, there is no standard care for older AML patients and there is no FDA approved drug therapies for those who can’t receive intensive chemotherapy. It’s believed that patients 55 or older are best managed with non-intensive therapies, because of intensive therapy related deaths estimated at 15% to 20%.

HMAs are being used as low intensity monotherapy for AML patients who are elderly or find standard induction therapy intolerable. However, HMAs that are used alone or in combination with venetoclax are not approved for treating AML.