Pharmaceutical Business review

FDA grants orphan drug status for AbbVie’s veliparib to treat advanced squamous 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.

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.

NSCLC is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.

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

Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie, said: "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.

"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.

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.

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.

There are three main subtypes of NSCLC: adenocarcinoma, squamous cell (epidermoid) carcinoma and large cell (undifferentiated) carcinoma.

Currently, about 25 to 30 percent of NSCLC cases worldwide are squamous tumors. Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.

About Veliparib

Veliparib is an investigational oral poly (adenosine diphosphate [ADP]–ribose) polymerase (PARP) inhibitor being evaluated in multiple tumor types.

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.

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.

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.

 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.