FDA Approves First 'Pre-Surgical' Drug for Breast Cancer

The U.S. Food and Drug Administration on Monday, September 30th 2013, approved the first drug to be used before the surgical treatment of breast cancer.

Perjeta (pertuzumab), which was granted accelerated approval by the FDA, is already used to treat breast cancer in its advanced stages. This is the first time that women with early-stage HER2-positive breast cancers -- a typically aggressive form of tumor -- will have the chance to start treatment with something other than surgery, the agency said. The hope is that pre-surgical treatment could result in less invasive surgeries and improved survival rates. Typically, surgery has been the first step in breast cancer treatment. "We are seeing a significant shift in the treatment paradigm for early-stage breast cancer," Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said in an agency statement released Monday. "By making effective therapies available to high-risk patients in the earliest disease setting, we may delay or prevent cancer recurrences.''

An FDA advisory panel had voted unanimously earlier this month to recommend approval of broader use of the drug. An agency report had said the drug looked promising in trials, but added that drug-related cardiac concerns required additional research. Perjeta is made by Roche Group's Genentech division; accelerated approvals usually are given to drugs that haven't been thoroughly tested but show promising results in early trials. Members of the advisory panel that voted on Perjeta earlier this month had insisted at the time that Genentech carry out more trials to ensure the drug will enable breast cancer patients to live longer, healthier lives. At the time, cancer specialists welcomed the news. "This is tremendously exciting," said Dr. Amy Tiersten, associate professor in the division of hematology and medical oncology at Mount Sinai Medical Center in New York City. "Pertuzumab was FDA approved in 2012 for the treatment of metastatic breast cancer when it was shown that it improved survival when added to standard regimens for HER2-positive metastatic breast cancer," Tiersten said. Metastatic breast cancer is cancer that has spread to other parts of the body. "But the really exciting information is when this can be translated into earlier stages of disease, where we can completely eradicate the disease and cure more women," she said.

The trial the panel vote was based on showed that when Perjeta was added to standard pre-surgical regimens for HER2-positive breast cancer, the chance of finding no cancer after the treatment was nearly doubled, Tiersten said. Women who have chemotherapy before surgery and who achieve complete remission -- no cancer at the time of breast surgery -- have a much greater chance of being completely cured of their disease, she said. Dr. Aye Moe Thu Ma, an attending physician in breast surgical oncology with St. Luke's and Roosevelt Hospitals in New York City, also voiced enthusiasm at the time. "We currently have limited options for [first-step] treatment of breast cancer," Ma said. "I'm excited that this may provide a more specific treatment for people with HER2-positive cancer." This means women may be able to keep their breasts because the tumor size is reduced before surgery, and some women may prefer this, she added. Both short- and long-term side effects will need to be examined to fully evaluate risks and benefits of this medication if it is approved, Ma added.

Perjeta is one of many newer biologic agents used to treat breast cancer. "These biologic, or 'targeted,' treatments work on breast cancer cells in a more specific way than some older treatments, so there are many fewer casualties to normal cells, translating into many fewer side effects for our patients," Tiersten explained. More than 4,800 women are enrolled in the trial the FDA accelerated approval was based on, and it will provide further data on efficacy, safety and long-term outcomes. Those results are expected in 2016, according to the agency. About one in eight women in the United States will develop breast cancer in her lifetime. According to the Mayo Clinic, HER2-positive breast cancer is a breast cancer driven by a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells. HER2-positive breast cancers typically are more aggressive than other types of breast cancer. But there are effective drug treatments, including trastuzumab (Herceptin) and lapatinib (Tykerb). Both drugs can produce side effects, including congestive heart failure, according to the Mayo Clinic.