Just a few weeks after a US regulatory panel backed expansion of its label, the Food and Drug Administration has approved Roche's Perjeta as the first preoperative breast cancer therapy to get the green light.

Specifically, the agency has granted accelerated approval to Perjeta (pertuzumab) as part of a complete treatment regimen for patients with early-stage breast cancer before surgery (neoadjuvant setting). The drug was approved last year for advanced or late-stage HER2-positive breast cancer in combination with Roche’s blockbuster Herceptin (trastuzumab) and docetaxel.

The expanded approval is based primarily on data from a 417-patient Phase II study showing that 39% of patients receiving the combination of Perjeta, Herceptin and docetaxel had no evidence of tumour tissue detectable at the time of surgery, known as a pathological complete response (pCR). This compared to about 21% of patients who received Herceptin plus docetaxel.

“We are seeing a significant shift in the treatment paradigm for early stage breast cancer,” said Richard Pazdur, 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."

As part of getting accelerated approval through the FDA's priority review programme, Roche needs to conduct a confirmatory trial which is being conducted in participants with HER2-positive breast cancer who had prior surgery and are at high risk of having their cancer return. More than 4,800 patients are enrolled and results are expected in 2016.

Roche notes that pCR is a common measure of neoadjuvant treatment effect in breast cancer and can be assessed more quickly than traditional endpoints. Chief medical officer Hal Barron noted that “a new approval pathway has made Perjeta available to people with HER2-positive early breast cancer several years earlier than previously possible [and] together with the FDA, we’ve charted new territory".