FDA Approves First Pre-Surgical Breast Cancer Drug

"Tiger face" is not currently listed on the known side-effects of Perjeta.

“Tiger face” is not currently listed on the known side-effects of Perjeta.

While many of the hundreds of thousands of people who are diagnosed each year with breast cancer go through radiation or chemotherapy before surgery, until now drugs aimed at treating breast cancer were not FDA-approved for use in the pre-surgery stage.

Today, the FDA granted accelerated approval to Perjeta, the brand-name for pertuzumab, a breast cancer treatment drug produced by Roche. Perjeta was approved last year for post-surgical treatment of some breast cancers, but this is the first time the FDA has signed off on a medication for use before going under the knife.

Perjeta will be used to treat patients with HER2-positive, early stage breast cancer (tumor greater than 2 cm in diameter or with positive lymph nodes) who are at high risk of having their cancer return, metastasize, or of dying from the disease. About 20% of breast cancer patients are HER2-positive.

The FDA based the approval on a study of 417 participants who were randomly assigned to receive one of four pre-surgical treatment regimens: Herceptin (trastuzumab) plus chemotherapy, Perjeta plus Herceptin and chemo, Perjeta plus Herceptin, or Perjeta plus chemo.

According to the results, about 39% of those who received the triple play of Perjeta, Herceptin and chemo were found to have no invasive cancer in the breast of lymph nodes. By comparison, only 21% of patients who received Herceptin plus chemo reached this same state.

The FDA has moved on to a confirmatory trial, looking at 4,800 patients 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, but some doctors are already prescribing Perjeta to pre-surgery patients.

But it won’t come cheaply. A for the manufacturer tells the AP that a combined regimen of Perjeta plus Herceptin would cost the patient between $27,000 and $49,000, for anywhere between nine and 18 weeks of treatment.

“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 . “By making effective therapies available to high-risk patients in the earliest disease setting, we may delay or prevent cancer recurrences.”

An estimated 232,340 women will be diagnosed with breast cancer this year, while nearly 40,000 people will die from the disease during the same time. It is the second-largest leading cause of cancer-related death in women.