The U.S. Food and Drug Administration today announced it will revoke the approval of Avastin for breast cancer, citing a lack of evidence that the benefits outweigh the risks.
Avastin was approved for metastatic breast cancer in February 2008 under the agency's accelerated approval program, which offers patients early access to promising drugs while confirmatory clinical trials are carried out.
"Unfortunately the additional studies failed to confirm Avastin's initial promise," FDA Commissioner Dr. Margaret Hamburg said today.
The additional studies, carried out by Avastin maker Genentech, found only a small effect on tumor growth and no evidence that patients lived longer than they would taking standard chemotherapy. Because the drug can cause severe high blood pressure, bleeding, heart failure and perforations in the nose, stomach and intestines, an FDA advisory panel in June recommended revoking its approval.
The recommendation triggered an outcry from breast cancer patients who say Avastin slowed their disease.
"I want every available weapon in my arsenal as I fight this devastating disease," Priscilla Howard, a metastic breast cancer patient who had been taking Avastin for more than two years ,urged the panel.
But without a reliable indicator of which women with breast cancer might benefit, Hamburg said she was unable to leave it on the market for that use.
"I feel deeply for these women who are seeking therapies that may work for them," she said, adding that she was speaking as a doctor and a woman. "Sometimes despite the hopes of investigators, patients, industry and even the FDA itself, the results of rigorous testing can be disappointing."
The drug will remain on the market for treatment of other cancers, which means breast cancer patients can use it off-label.
"If an individual patient sits down with their physician and goes over the risks and benefits and wants to continue, that is a decision that they can make," said Hamburg.
However, the FDA decision could free up insurers to deny coverage of the drug, which can cost up to $100,000 a year. In June, a spokesman for the Centers for Medicare and Medicaid said Medicare would continue to cover Avastin for breast cancer treatment even if the FDA withdrew its approval.
A spokeswoman for Genentech said the decision was disappointing, but insisted the company would continue working to optimize Avastin and identify breast cancer patients most likely to benefit.
"Despite today's action, we will start a new Phase III study of Avastin in combination with paclitaxel in previously untreated metastatic breast cancer and will evaluate a potential biomarker that may help identify which people might derive a more substantial benefit from Avastin," a company spokeswoman said in a statement today. (Paclitaxel is also a chemotherapy drug.)
Hamburg welcomed Genentech's effort and said she looks forward to working with them on Avastin.
The FDA has approved 39 drugs through the accelerated approval program. When asked how many accelerated approvals had been revoked, Hamburg said she was unsure.
ABC News' Lara Salahi and Brian Hartman contributed to this report.