Rare Disease Report

FDA Grants Breakthrough Therapy Designation to AML Therapy

JANUARY 27, 2016
Rebekah Harrison
The FDA granted AbbVie breakthrough therapy designation for venetoclax in combination with hypomethylating agents (HMAs) for the treatment of patients with untreated acute myeloid leukemia (AML) who are ineligible to receive standard induction therapy.
Venetoclax is an inhibitor of the B-cell lymphoma-2 (BCL-2) protein being developed by AbbVie in partnership with Genentech and Roche.

What is Acute Myeloid Leukemia?

Acute myeloid leukemia is a type of cancer in which the bone marrow makes abnormal myeloblasts. Symptoms include fatigue, recurrent infections and easy bruising. Acute myeloid leukemia is generally a disease of older people and is uncommon before the age of 45. The average age of a patient with AML is about 67 years. The number of new cases of acute myeloid leukemia was 4.0 per 100,000 men and women per year.
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. 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. There is no FDA-approved treatment for patients not eligible to receive chemotherapy however, there are numerous orphan drugs approved for this rare cancer (i.e., filgastim, idarubicin, sargramostim).
The Breakthrough Therapy Designation is given if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints.
The designation provides Abbvie with more input from the FDA during the developmental process as well as the possibility of submitting a rolling review.
"Acute myeloid leukemia 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," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "This third Breakthrough Therapy Designation for venetoclax highlights our efforts to pursue the significant and broad potential of this therapy."


Venetoclax Receives 3rd Breakthrough Therapy Designation from the FDA for the Combination Treatment of Patients with Untreated Acute Myeloid Leukemia not Eligible for Standard Induction Chemotherapy [news release]. Chicago, IL; AbbVie: January 28, 2016. http://www.prnewswire.com/news-releases/venetoclax-receives-3rd-breakthrough-therapy-designation-from-the-fda-for-the-combination-treatment-of-patients-with-untreated-acute-myeloid-leukemia-not-eligible-for-standard-induction-chemotherapy-300211208.html

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.