FDA Approves Perjeta for Pre-Operative Breast Cancer

The US Food and Drug Administration (FDA) has approved the targeted drug Perjeta (pertuzumab) for certain women with early stage breast cancer before surgery. This is the first time the FDA has approved a drug for treatment of breast cancer before surgery.

The approval is specifically for women with HER2-positive, locally advanced, inflammatory or early stage breast cancer who are at high risk of having their breast cancer return or spread, or are at high risk of dying from the disease. The drug is meant to be used in combination with another targeted drug and with chemotherapy.

HER2-positive describes a type of breast cancer that has increased amounts of the HER2 protein, which contributes to cancer cell growth and survival. About 1 out of 5 breast cancers are HER2-positive. These cancers tend to grow and spread more aggressively than other types of breast cancer. Perjeta works by blocking the proteins’ growth signals.

“We are seeing a significant shift in the treatment paradigm for early stage breast cancer,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a statement. “By making effective therapies available to high-risk patients in the earliest disease setting, we may delay or prevent cancer recurrences.’’

The FDA acted under its accelerated approval program for drugs that show early promise against a serious disease. The company that makes the drug must submit additional clinical information after approval to demonstrate its benefit.

The FDA first approved Perjeta in 2012 to treat women with HER2-positive breast cancer that had spread to other parts of the body.

Perjeta’s accelerated approval for pre-surgery treatment is based on a study designed to look at the disappearance of invasive cancer in the breast and lymph nodes before surgery. In the study, 417 women were randomly assigned to receive 1 of 4 drug combinations:

  • the targeted drug Herceptin (trastuzumab) plus the chemotherapy drug docetaxel
  • Perjeta plus Herceptin and docetaxel
  • Perjeta plus Herceptin
  • Perjeta plus docetaxel

About 39% of those who received Perjeta plus Herceptin and docetaxel achieved the absence of invasive cancer in the breast and lymph nodes, compared to about 21% who received trastuzumab plus docetaxel. The study did not show if the three-drug combination helped keep the cancer from coming back better than the other combinations, or if it helped the women live longer.

A study to confirm the accelerated approval includes 4,800 participants with HER2-positive breast cancer who had prior breast cancer surgery and are at high risk of having their cancer return. Results are expected in 2016.

The most common side effects reported from Perjeta plus Herceptin and docetaxel were hair loss, diarrhea, nausea and a decrease in infection-fighting white blood cells. Other significant side effects included decreased heart function, and allergic reactions.

Perjeta is marketed by Genentech.

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