FDA Approves Pertuzumab for Some Early-Stage HER2-Positive Breast Cancers

Pertuzumab can be added to trastuzumab and chemotherapy for people at high risk of recurrence
Breast Cancer News
February 14, 2018
Eric Fitzsimmons
Reviewed By: 
Shanu Modi, MD

The FDAinfo-icon has approved the use of pertuzumabinfo-icon (Perjeta) given with trastuzumabinfo-icon (Herceptininfo-icon) and chemotherapyinfo-icon for people with early-stageinfo-icon, HER2-positive breast cancer who have a higher risk of recurrenceinfo-icon.

The results were based on the APHINITY trial that showed a slight benefit overall but somewhat stronger results for people who had cancers that were hormone receptorinfo-icon-negative or had cancer found in their lymphinfo-icon nodes.


The APHINITY trial was a phase III, double-blind study designed to see if people with early-stage, HER2-positive breast cancer would benefit from adding pertuzumab to the current standard treatment of chemotherapy and a year of trastuzumab. There were 4,804 people included in the trial randomized to one of two treatment arms:

  • 2,400 got chemotherapy with 1 year of trastuzumab and pertuzumab.
  • 2,404 got chemotherapy with 1 year of trastuzumab and a placeboinfo-icon.

Maker Genentech first announced in March that the APHINITY trial had met its primary endpointinfo-icon, showing that there was a lower risk of a recurrence of invasive (stage I or higher) breast cancer or death when taking pertuzumab. But the data were not shared until the American Society of Clinical Oncology meeting in June. The study findings showed a benefit for all study participants that was statistically significantinfo-icon — meaning it was likely not due to chance — but also small. In 3 years of follow up, the study found

  • 7.1 percent of people taking pertuzumab had a recurrence or died
  • 8.7 percent of people taking the placebo had a recurrence or died

Pertuzumab costs more than what people are already paying for HER2-positive treatment, and the improvement in risk of recurrence or death was less than two percentage points. Doctors at ASCO questioned if they could justify recommending adding nearly $100,000 for treatment that would help such a small percentage of people.

But when researchers looked only at people who had a high risk of recurrence, the data showed a larger improvement from adding pertuzumab.

Looking just at people with hormone receptor-negative breast cancer, which is more likely to recur than other types of breast cancer

  • 8.2 percent of those taking pertuzumab had a recurrence or died
  • 10.6 percent of those taking the placebo had a recurrence or died

The difference was even larger for those who had breast cancer found in the lymph nodes, sometimes referred to as node-positiveinfo-icon disease:

  • 9.2 percent of those taking pertuzumab had a recurrence or died.
  • 12.1 percent of those taking the placebo had a recurrence or died.

The most common side effects for the pertuzumab group were diarrheainfo-icon, low white blood cellinfo-icon counts, tiredness and nauseainfo-icon.

The FDA also granted regular approval  for pertuzumab (in combination with trastuzumab and chemotherapy) in neoadjuvant treatment for certain HER2-positive breast cancers. The neoadjuvant use of pertuzumab had been given accelerated approval in 2013, and these further results in early-stage breast cancerinfo-icon completed the required follow up for regular approval. These breast cancers were either inflammatory, locally advanced, had a tumorinfo-icon of more than 2 centimeters, or were node-positive.

What This Means for You

Medicines like trastuzumab have greatly lowered the risk of recurrence for early-stage, HER2-positive breast cancers. But certain other features, such as cancer found in the lymph nodes or being HER2-positive and hormone receptor-negative disease, still face a somewhat higher risk. And until now, people with these higher-risk features got the same treatment as people with average risk.

Pertuzumab provides a treatment option to add to standard treatment for people with HER2-positive disease and who are at higher risk for recurrence. The addition of pertuzumab further lowers the risk of a recurrence, with relatively few side effects, but a high financial cost. Speak to your doctor about the risk of recurrence for your diagnosisinfo-icon, financial impacts, and which treatment would be right for you. 

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