AbbVie, a global, research-based biopharmaceutical company, announced its investigational medicine venetoclax, an inhibitor of the B-cell lymphoma-2 (BCL-2) protein that is being developed in partnership with Genentech and Roche, has been granted breakthrough therapy designation by the US FDA for the treatment of chronic lymphocytic leukemia (CLL) in previously treated (relapsed/refractory) patients with the 17p deletion genetic mutation.

CLL is a slow-progressing cancer of the bone marrow and blood in which the bone marrow makes too many lymphocytes, a type of white blood cell. CLL accounts for approximately one quarter of the new cases of leukemia diagnosed in the United States. Approximately 3-10 per cent of CLL patients have 17p deletion at diagnosis, and it occurs in 30-50 per cent of patients with relapsed/refractory CLL. 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 17p deletion is less than 2-3 years.

"The breakthrough therapy designation of venetoclax supports the continued development of this investigational medicine in CLL patients with 17p deletion," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "The continuing advancement of the venetoclax development programme is one example of AbbVie's focus on delivering innovative medicines that address unmet clinical needs."

According to the FDA, breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation includes preliminary clinical evidence demonstrating a drug may have substantial improvement on at least one clinically significant endpoint compared to available therapy. A breakthrough therapy designation conveys all of the fast track programme features, as well as more intensive FDA guidance on an efficient drug development programme.

Venetoclax is an investigational oral B-cell lymphoma-2 (BCL-2) inhibitor being evaluated for the treatment of patients with various cancer types. The BCL-2 protein prevents apoptosis of some cells, including lymphocytes, and can be expressed in some cancer types.

Venetoclax is designed to selectively inhibit the function of the BCL-2 protein. Venetoclax is being developed in collaboration with Genentech and Roche. Together, the companies are committed to BCL-2 research with venetoclax, which is currently being evaluated in phase 2 and phase 3 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.