According to the Los Angeles County Department of Public Health, breast cancer is the second most common cause of premature death, behind coronary heart disease, for women in the county. New drugs to treat breast cancer are moving down the Food and Drug Administration (FDA) pipeline. Last year, Perjeta, a drug manufactured by Genentech, gained FDA approval for treatment of late-stage breast cancer. On September 12, a FDA advisory committee cleared the way for the first approval of a Perjeta for the treatment of women before surgery to remove their tumors. The committee voted 13 to 0, with one abstention in favor of the expanded use of Perjeta.
The FDA noted that pre-surgery treatment with the drug might help make some inoperable tumors amenable to surgery, or make tumors small enough to allow for breast-conserving surgery rather than complete removal of the breast. In addition, the approval could serve as a model for a new path to quicker approvals of cancer drugs. “This is a historic moment,” noted Dr. Mikkael A. Sekeres, an associate professor at the Cleveland Clinic who served as the chairman of the advisory committee. He added, “We are supporting the rapid movement of a highly active drug for metastatic cancer to the first-line setting, with the hope that women with earlier stages of breast cancer will live longer and better.”
Perjeta still requires formal FDA approval for the new use; however, agency officials indicated it was likely to do so. Perjeta is approved to treat only so-called HER2-positive tumors, which account for about 20% of all breast cancers. Pre-surgery treatment would be approved for only a minority of those patients, with large tumors or other features that increase the risk. Genentech estimates that 15,000 American women a year would be eligible for preoperative treatment with Perjeta, out of about 220,000 new cases of early-stage breast cancer.
Preoperative drug treatment is known as neoadjuvant therapy. Genentech’s main neoadjuvant trial involved only about 400 patients treated for only 12 weeks. About 39.3% of patients who received Perjeta plus Herceptin and taxotere had what was called a complete pathologic response, meaning no invasive cancer was detected in their breast tissue or in any removed lymph nodes. For patients who received only Herceptin and taxotere, only 21.5%. If Perjeta attains FDA approval, it would be the first neoadjuvant drug approval for any cancer, not just breast cancer. Thus, it is likely to facilitate insurance reimbursement. That is a big deal because treating a patient with Herceptin and Perjeta together for 9 to 18 weeks before surgery would cost $27,000 to $49,000, according to Genentech. In addition, this preoperative treatment would not eliminate the need for drugs after surgery.