Roche Holding AG’s Perjeta won the backing of U.S. regulatory advisers to potentially become the first pre-surgical drug treatment for breast cancer that could lead to a less-invasive procedure.
The benefits of expanding use of Perjeta to early-stage breast cancer patients outweigh the risks, advisers to the Food and Drug Administration voted 13-0 today in Silver Spring, Maryland. The drug was approved last year for patients with HER2-positive advanced breast cancer that has spread.
Expanded clearance would allow treatment with Perjeta as an initial, or neoadjuvant, step before surgery that may shrink tumors. That could lead to a less-invasive surgical procedure. The FDA is pushing approval of early breast-cancer therapies based on suggestive benefit, a move that may bring treatments to patients several years faster than older methods.
Mikkael Sekeres, chairman of the advisory panel and a cancer doctor at the Cleveland Clinic, called the vote “a historic moment.”
“We are supporting the rapid movement of a highly active drug in the metastatic setting to an earlier setting with the hope that women who use this drug will live longer,” he said.
The FDA may decide by the end of October whether to approve Perjeta’s wider use.
Perjeta, first approved for advanced breast cancer in June 2012, may generate sales of $1.9 billion in 2016, according to the average of five analysts’ estimates compiled by Bloomberg. Roche’s Genentech unit estimates about 15,000 women with HER2-positive early stage breast cancer could receive neoadjuvant treatment with Perjeta each year.
The panel’s “recommendation is a step toward bringing Perjeta to people with HER2-positive early stage breast cancer, where treatment can potentially prevent the disease from returning and spreading,” Hal Barron, Roche’s chief medical officer, said in a statement.
Cancer drugs often are approved for early stages of a disease several years after the treatments are cleared for advanced stage. Roche’s Herceptin was approved in 1998 for metastatic breast cancer, then in 2006 for early forms of the disease. Perjeta could be approved for earlier use just more than a year after original clearance under a new FDA policy that bases accelerated approval on tumor shrinkage.
This is a “significant quantum leap,” Richard Pazdur, director of the Office of Hematology & Oncology Products in the FDA’s Center for Drug Evaluation and Research, said at the meeting.
The FDA released a draft proposal in May 2012 guiding companies to use tumor eradication as a study goal to gain accelerated approval for neoadjuvant breast cancer treatment.
Roche is seeking accelerated approval of Perjeta based on mid-stage trials that showed “statistically significant improvements” in eradication of tumor tissue, FDA staff said in a Sept. 10 report. The drug would be cleared for sale on the condition the company conduct trials after the approval proving Perjeta extends disease-free survival.
The Basel, Switzerland-based company is doing a final-phase trial called Aphinity measuring disease-free survival that could confirm the benefit of the drug, FDA staff said. Roche expects data on disease-free survival from the trial in 2016, Susan Willson, a Genentech spokeswoman, said.
Roche studied Perjeta’s ability to eradicate tumors when used with its older breast-cancer drug Herceptin and chemotherapy. The majority of people with HER2-positive early stage breast cancer who receive neoadjuvant treatment take a combination of Herceptin and chemotherapy, Willson said. HER2-positive breast cancer tends to be more aggressive than other forms.
Herceptin brought in revenue of $6.3 billion last year. Roche is also studying another treatment, Kadcyla, in the neoadjuvant setting.