FDA grants accelerated approval of Perjeta for people with HER2-positive early stage breast cancer

The U.S. Food and Drug Administration (FDA) granted accelerated approval of a Perjeta- (pertuzumab) regimen for neoadjuvant treatment (use before surgery) in people with high-risk, HER2-positive early stage breast cancer.

This approval is based primarily on data from a Phase II study showing that nearly 40 percent of people receiving the combination of Perjeta, Herceptin- (trastuzumab) and docetaxel chemotherapy had no evidence of tumor tissue detectable at the time of surgery (known as a pathological complete response, or pCR). The most common severe (Grade 3 or higher) AEs for the Perjeta regimen were neutropenia (decrease in a certain type of white blood cell, 44.9 percent), febrile neutropenia (fever associated with decrease in a certain type of white blood cell, 8.4 percent), leukopenia (decrease in overall white blood cells, 4.7 percent) and diarrhea (5.6 percent).

This new neoadjuvant indication for Perjeta is for use prior to surgery in combination with Herceptin and docetaxel chemotherapy in people with HER2-positive, locally advanced, inflammatory, or early stage (tumor is greater than two centimeters in diameter or node positive) breast cancer. Perjeta should be used as part of a complete treatment regimen for early stage breast cancer. This use of Perjeta is based on an improvement in the percentage of people who had no evidence of cancer in the breast or lymph nodes at the time of surgery. Currently, no data have shown whether or not treatment with Perjeta prior to surgery improves survival. The safety of Perjeta as part of a doxorubicin (chemotherapy)-containing regimen has not been established. The safety of Perjeta administered for greater than six cycles for early stage breast cancer has not been established.

Background on Neoadjuvant Treatment:

Upon receiving an early stage breast cancer diagnosis, a person typically undergoes surgery and may then receive a medicine in the "adjuvant" (post-surgery) setting. Neoadjuvant treatment is another treatment option given after diagnosis but before surgery. Neoadjuvant treatment may allow a doctor to quickly access whether a medicine is working, and may also reduce a tumor's size so it is easier to surgically remove.

WHY THIS APPROVAL IS IMPORTANT

• Treating people with breast cancer early, before the cancer has spread, may offer the best chance of preventing the disease from returning.
• The Perjeta regimen is the first treatment approved under a new FDA pathway for neoadjuvant use in breast cancer.
• This new approval pathway makes Perjeta available to people with high-risk, early stage breast cancer more quickly than is possible with traditional approvals.
• HER2-positive breast cancer is a particularly aggressive form of the disease that affects approximately 25 percent of people with breast cancer.