AbbVie Receives EMA Orphan Drug Designation for Investigational Compound Venetoclax for the Treatment of Acute Myeloid Leukemia (AML)

- FDA also recently granted venetoclax Breakthrough Therapy Designation (BTD) and Orphan Drug Designation (ODD) for the treatment of patients with AML

- AML is the most common type of acute leukemia in adults

News provided by

AbbVie

Feb 26, 2016, 06:00 ET

NORTH CHICAGO, Ill., Feb. 26, 2016 /PRNewswire/ -- AbbVie (NYSE:ABBV), a global biopharmaceutical company, today announced the European Medicines Agency (EMA) has granted Orphan Drug Designation to venetoclax, an investigational, oral B-cell lymphoma-2 (BCL-2) inhibitor, for the treatment of acute myeloid leukemia (AML). AML can be a rapidly progressing cancer of the blood and bone marrow and is the most common type of acute leukemia in adults.¹ Venetoclax is being developed by AbbVie in partnership with Genentech and Roche.

AML is generally a disease of older people and is uncommon before the age of 45, with the average age being 67 years of age.² In AML, the body produces too many of a specific type of white blood cell (myeloblast), which can crowd out healthy blood cells.¹ In Europe, the annual incidence rate of AML is estimated to be 1/33,000-1/25,000.³

The EMA previously granted Orphan Drug Designation to venetoclax for the treatment of chronic lymphocytic leukemia (CLL). Orphan Designation is granted to therapies aimed at the treatment, prevention or diagnosis of life-threatening diseases that affect no more than five in 10,000 persons in the European Union (EU) and for which no satisfactory therapy is available. The medicine must also provide significant benefit to those affected by the condition.⁴

"There have been very few treatment advances for patients with AML who are older than 60, the patient population that is most often affected by this aggressive and life-threatening cancer," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "These designations for venetoclax not only underscore the unmet need for patients with AML, but also reinforce our ongoing commitment to battling cancer and researching scientific advances in oncology."

The U.S. Food and Drug Administration recently granted venetoclax both Breakthrough Therapy Designation (BTD) and Orphan Drug Designation (ODD) for the treatment of patients with AML. The FDA has also granted venetoclax Breakthrough Therapy Designation for the treatment of CLL in previously treated (relapsed/refractory) patients with the 17p deletion genetic mutation and in combination with rituximab for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL). Additionally, venetoclax recently received validation from the EMA for its Marketing Authorization Application (MAA) for the treatment of CLL patients with 17p deletion or TP53 mutation, as well as acceptance by Health Canada for the New Drug Submission (NDS) for the treatment of patients with CLL who have received at least one prior therapy, including patients with 17p deletion.

About Venetoclax
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 (programmed cell death) of some cells, including lymphocytes, and can be over 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 3 clinical trials for the treatment of relapsed/refractory CLL, along with studies in several other cancers. Venetoclax is an investigational compound and its safety and efficacy have not been evaluated by the EMA or any other health authority. Additional information regarding the venetoclax clinical trials is available on www.clinicaltrials.gov.

About AbbVie Oncology
AbbVie's oncology research is focused on the discovery and development of targeted therapies that work against the processes cancers need to survive. By investing in new technologies and approaches, AbbVie is breaking ground in some of the most widespread and difficult-to-treat cancers, including glioblastoma multiforme, multiple myeloma and chronic lymphocytic leukemia. AbbVie's oncology pipeline includes multiple new molecules in clinical trials being studied in more than 15 different cancers and tumor types. For more information on AbbVie Oncology, please visit http://oncology.abbvie.com.

About AbbVie
AbbVie is a global, research-based biopharmaceutical company formed in 2013 following separation from Abbott Laboratories. The company's mission is to use its expertise, dedicated people and unique approach to innovation to develop and market advanced therapies that address some of the world's most complex and serious diseases. Together with its wholly-owned subsidiary, Pharmacyclics, AbbVie employs more than 28,000 people worldwide and markets medicines in more than 170 countries. For further information on the company and its people, portfolio and commitments, please visit www.abbvie.com. Follow @abbvie on Twitter or view careers on our Facebook or LinkedIn page.

Forward-Looking Statements
Some statements in this news release may be forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry.

Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," in AbbVie's 2014 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.

¹ MedlinePlus, U.S. National Library of Medicine (2015). "Acute Myeloid Leukemia." https://www.nlm.nih.gov/medlineplus/acutemyeloidleukemia.html. Accessed January 21, 2016.
² American Cancer Society (2016). "Leukemia—Acute Myeloid (Myelogenous)." http://www.cancer.org/cancer/leukemia-acutemyeloidaml/detailedguide/leukemia-acute-myeloid-myelogenous-key-statistics. Accessed January 27, 2016.
³ OrphaNet (2014). "Acute myeloid leukemia." http://www.orpha.net/consor4.01/www/cgi-bin/OC_Exp.php?lng=EN&Expert=519 Accessed January 27, 2016.
⁴ European Medicines Agency (2015). "Orphan Designation." http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp. Accessed January 27, 2016.