AbbVie Receives FDA Orphan Drug Designation for Investigational Medicine Veliparib for the Treatment of Advanced Squamous Non-Small Cell Lung Cancer

- Veliparib in combination with chemotherapy or radiation is being investigated in ongoing studies for advanced squamous non-small cell lung cancer (NSCLC)[1]

- Squamous cell carcinoma accounts for about 25 to 30 percent of NSCLC cases[2]

- Lung cancer is the leading cause of cancer-related deaths in the U.S.[2]

News provided by

AbbVie

Nov 04, 2016, 01:33 ET

NORTH CHICAGO, Ill., Nov. 4, 2016 /PRNewswire/ -- AbbVie (NYSE: ABBV), a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to veliparib, an oral poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) inhibitor, being investigated in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC).

PARP is a naturally-occurring enzyme in the body that repairs damage to DNA in cells. While this repair is a useful process to maintain the integrity of healthy cells, the same process may also help repair DNA in cancer cells, causing them to survive.³ Researchers are investigating whether veliparib, a PARP inhibitor, in combination with DNA damaging therapies, such as chemotherapy or radiation, may lessen the repair of DNA damage in cancer cells, eventually causing some cells to die.³

NSCLC is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.² Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.² Squamous cell carcinoma accounts for about 25 to 30 percent of NSCLC cases.² It is usually found in the middle airways of the lungs and is often linked to a history of smoking.²

"Lung cancer is the leading cause of cancer-related deaths in the U.S. and can be difficult to treat, particularly when diagnosed in later stages. This Orphan Drug Designation for veliparib recognizes the significant unmet need in patients with advanced squamous non-small cell lung cancer," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "AbbVie is committed to the ongoing development of veliparib in solid tumors to help advance the care of people living with cancer."

AbbVie is currently investigating the efficacy and safety of veliparib in combination with chemotherapy or radiation for the treatment of advanced squamous NSCLC, including in Phase 3 studies. Veliparib is not currently approved to treat any form of NSCLC.¹

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.⁴

About Non-Small Cell Lung Cancer
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.² There are three main subtypes of NSCLC: adenocarcinoma, squamous cell (epidermoid) carcinoma and large cell (undifferentiated) carcinoma.² Currently, about 25 to 30 percent of NSCLC cases worldwide are squamous tumors.² Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.²

About Veliparib
Veliparib is an investigational oral poly (adenosine diphosphate [ADP]–ribose) polymerase (PARP) inhibitor being evaluated in multiple tumor types.^5,6 PARP is a naturally-occurring enzyme in the body that repairs damage to DNA in cells. While this repair is a useful process to maintain the integrity of healthy cells, the same process may also help repair DNA in cancer cells, causing them to survive.³

Discovered and developed by AbbVie researchers, veliparib is being studied in combination with chemotherapy or radiation to help determine whether it can prevent DNA repair in cancer cells to possibly increase the effectiveness of common DNA-damaging therapies, such as chemotherapy or radiation.⁵ Veliparib is currently being studied in more than a dozen cancers, including in Phase 3 studies in advanced squamous and non-squamous non-small cell lung cancer (NSCLC), ovarian cancer and breast cancer.⁷ Veliparib is an investigational medicine and its efficacy and safety have not been established by the U.S. Food and Drug Administration (FDA) or any other health authority.

About AbbVie in Oncology
AbbVie is striving to outsmart cancer by working with scientists, physicians, industry peers, patient advocacy groups and most importantly patients, to discover, develop and provide new therapies that will have a remarkable impact on the lives of people around the world affected by cancer. Our goal is to provide medicines that make a transformational improvement in cancer treatment and outcomes for cancer patients. By exploring and investing in new pathways, technologies and approaches, AbbVie is breaking ground in some of the most widespread and difficult-to-treat cancers. We are also exploring solutions to help patients obtain access to our cancer medicines. With the acquisition of Pharmacyclics in 2015 and Stemcentrx in 2016, and through several collaborations, AbbVie's oncology portfolio consists of marketed medicines and a pipeline containing multiple new molecules being evaluated worldwide in nearly two hundred clinical trials in 20 different tumor types. For more information about AbbVie Oncology, please visit http://abbvieoncology.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 our Facebook and LinkedIn pages.

AbbVie 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 2015 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] ClinicalTrials.gov (2016). NCT02106546. https://clinicaltrials.gov/ct2/show/NCT02106546?term=veliparib&cond=lung+cancer&rank=7. Accessed October 2016.
[2] American Cancer Society (2016). Lung Cancer (Non-Small Cell). http://www.cancer.org/acs/groups/cid/documents/webcontent/003115-pdf.pdf. Accessed October 2016.
[3] Plummer ER et al. Targeting Poly (ADP-Ribose) Polymerase: A Two-Armed Strategy for Cancer Therapy. Clin Cancer Res. 2007;13(21): 6252-6256.
[4] U.S. Food & Drug Administration (2016). Developing Products for Rare Diseases & Conditions. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm. Accessed October 2016.
[5] Palma JP et al. ABT-888 confers broad in vivo activity in combination with temozolomide in diverse tumors. Clin Cancer Res. 2009;15(23):7277-7290.
[6] Anders CK et al. Poly (ADP-Ribose) polymerase inhibition: "targeted" therapy for triple-negative breast cancer. Clin Cancer Res. 2010;16(19):4702-4710.
[7] ClinicalTrials.gov (2016). Results: Veliparib: Open Studies. https://clinicaltrials.gov/ct2/results?term=veliparib&recr=Open. Accessed October 2016.