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FDA Grants ‘Breakthrough Therapy’ Designation for Leukemia Drug Venetoclax

Studies under way in patients with CLL

The investigational medication venetoclax (AbbVie/Genetech/Roche), an inhibitor of the B-cell lymphoma-2 (BCL-2) protein, has been granted “breakthrough therapy” status by the FDA for the treatment of chronic lymphocytic leukemia (CLL) in previously treated (relapsed/refractory) patients with the 17p deletion genetic mutation.

CLL accounts for approximately 25% of the new cases of leukemia diagnosed in the U.S. Approximately 3% to 10% of CLL patients have the 17p deletion at diagnosis, and this deletion can occur in 30% to 50% of patients with relapsed/refractory disease. The 17p deletion mutation is a genomic alteration in which a part of chromosome 17 is absent. The median life expectancy for CLL patients with the 17p deletion is less than 2 to 3 years.

According to the FDA, the “breakthrough therapy” designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for this designation include preliminary clinical evidence demonstrating that a drug may offer substantial improvement on at least one clinically significant endpoint compared with available therapies.

Venetoclax is an investigational oral BCL-2 inhibitor being evaluated for the treatment of patients with various types of cancer. The BCL-2 protein prevents the apoptosis of some cells, including lymphocytes, and can be expressed in cancer. Venetoclax is designed to selectively inhibit the function of the BCL-2 protein.

The drug is currently being evaluated in phase II and phase III clinical trials for the treatment of CLL, along with studies in several other cancers.

Venetoclax is an investigational compound, and its safety and efficacy have not been evaluated by the FDA or any other health authority.

Sources: AbbVie; May 6, 2015; and AbbVie; December 7, 2014.

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