The US Food and Drug Administration (FDA) granted accelerated approval to Roche and Genentech's pertuzumab for neoadjuvant breast cancer, a move towards earlier use of cancer drugs.

The lowdown: Oncologists are increasingly thinking about using anticancer agents earlier on, arguing that pre-surgery tumour debulking and early treatment can improve long-term outcomes. Roche and Genentech have now snagged a first regulatory approval to use their pertuzumab in just such a setting, in early-stage breast cancer before surgery.

“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 at the FDA in a statement.

Pertuzumab — a monoclonal antibody (mAb) that binds to HER2, preventing receptor dimerization — was first approved last year for use in combination with the HER2-targeted mAb trastuzumab and docetaxel chemotherapy in patients with HER2-positive metastatic breast cancer. The new accelerated approval is based on a 417-patient trial showing that neoadjuvant pertuzumab plus trastuzumab plus docetaxel induced a pathological complete response (pCR) in 39% of patients, whereas neoadjuvant trastuzumab plus docetaxel only induced a pCR in 21% of patients. Trastuzumab and docetaxel are used off-label in the neoadjuvant setting.

The approval was made possible in part by guidance the FDA issued last year on pCR, which is a fast surrogate end point that looks at the absence of invasive cancer in the breast and lymph nodes at surgery.

Trials are underway to identify other neoadjuvant breast cancer drugs. The adaptive I-SPY 2 trial, for example, is testing multiple agents in women with newly diagnosed locally advanced breast cancer.

Len Lichtenfeld, chief medical officer of the American Cancer Society, says that interest in neoadjuvant drug development extends to other cancer indications as well, including pancreatic cancer, liver cancer and ovarian cancer. “I don't think this drug approval is going to influence the further investigation of neoadjuvant treatments; it is the results of neoadjuvant use that are already influencing the community,” he adds.