FDA
13th September 2013

FDA Panel Backs Approval of Perjeta for Early-Stage Breast Cancer

The Perjeta regimen would be given as part of a broad treatment program that would inc

FDA Panel Backs Approval of Perjeta for Early-Stage Breast Cancer

Yesterday, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 13-0, with one abstention, in favor of approving the monoclonal antibody pertuzumab (Perjeta) as an neoadjuvant treatment for HER2-positive, early-stage breast cancer in combination with trastuzumab (Herceptin) and docetaxel (Taxotere) to help shrink tumors prior to surgery in the hope that it will make them easier to remove. Roche Holding AG’s Perjeta was approved last June for use in combination with trastuzumab and docetaxel for patients with HER2-positive metastatic breast cancer who haven’t received prior treatment with anti-HER2 therapy or chemotherapy.

The Perjeta regimen would be given as part of a broad treatment program that would include the chemotherapies docetaxel and either fluorouracil, epirubicin, and cyclophosphamide or carboplatin.

Panel chairman Dr. Mikkael Sekeres called the vote “a historic moment.”

“We are supporting the movement of a highly active drug for metastatic breast cancer to the first-line setting, with the hope that women with earlier stages of breast cancer will live longer and better,” said Sekeres, an associate professor of medicine at the Cleveland Clinic.

Breast cancer is the second deadliest form of cancer in U.S. women, and is expected to kill more than 39,000 Americans this year, according to the National Cancer Institute. More than 232,000 women are expected to be diagnosed with the disease during that time. About 6,000 to 8,000 deaths per year are attributed to the HER-2 form of the disease. However, breast cancer is highly treatable when detected early.

A randomized trial of 417 patients (click HERE to view summary of trial) showed patients receiving pertuzumab, trastuzumab, and docetaxel before surgery improved cPR by 39.3% (95% CI 30.0%-49.2%) compared with 21.5% (95% CI 14.1%-30.5%) in patients who only received trastuzumab and docetaxel. 

Panelists concluded that the totality of this trial’s evidence, including the drug’s relative safety in advanced breast cancer, tilted their decision in favor of approval despite the small trial size.

Much of the panel’s discussion focused on the measure used to judge the trial’s success – known as complete pathological response (cPR). The FDA defines cPR as the absence of tumor tissue at the time of surgery, and Perjeta would be the first drug to be approved based on the measure. This is a relatively weak measure on which to base an approval as there is no proof that if a patient achieves cPR she will live longer, or that her quality of life will improve.

Though more patients who took Perjeta had a cPR than those who did not, it was unclear whether those patients would have had better outcomes even without treatment.

The FDA has expressed some concern that Perjeta, in combination with anthracycline chemotherapies such as epirubicin, could cause heart problems, including left ventricular dysfunction. Roche said it intends to address the questions with an additional trial of Perjeta in combination with a variety of chemotherapy regimes, including anthracyclines. In addition, multiple panel members noted the need for strong labeling to restrict the agent’s use in only the appropriate population and not one in which it hasn’t been proven effective.

The company is conducting a follow-up trial using 4,800 patients who have already had surgery, as requested by the FDA. Panelists urged the FDA to carefully scrutinize the results of this follow-up trial to ensure they support the benefit to patients suggested by the small trial.

A decision from the agency is due Oct. 31. if approved, Perjeta would become the first agent to receive this indication from the FDA.

The FDA isn’t obligated to follow the advice of its advisory committees but usually does.

 

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