FDA Commissioner Dr. Margaret A. Hamburg said she will be revoking the agency’s approval of the breast cancer indication for Avastin, today, after the drug has not shown itself to be safe and effective for that use.

The drug will still remain an approved treatment on the market for colon, lung, kidney and brain cancer.

"This was a difficult decision. FDA recognizes how hard it is for patients and their families to cope with metastatic breast cancer and how great a need there is for more effective treatments. But patients must have confidence that the drugs they take are both safe and effective for their intended use," Hamburg said.

Hamburg says that women who take Avastin for metastatic breast cancer, risk potentially life-threatening side effects.

"After reviewing the available studies it is clear that women who take Avastin for metastatic breast cancer risk potentially life-threatening side effects without proof that the use of Avastin will provide a benefit, in terms of delay in tumor growth that would justify those risks. Nor is there evidence that use of Avastin will either help them live longer or improve their quality of life."

The risks include severe high blood pressure, bleeding and hemorrhaging, heart attack or heart failure, and the development of perforations in different parts of the body such as the nose, stomach, and intestines.

Avastin, approved in February 2008 for metastatic breast cancer under the FDA's accelerated approval program that allows a drug to be approved based on data that is not sufficiently complete to permit full approval.

The program allows earlier patient access to new drugs that are believed to treat serious or life-threatening conditions while clinical trials are conducted to confirm the effectiveness and safety of the drug.

If the clinical trials do not confirm that the drug is effective and safe for the patients and does not justify continued approval of the drug or drug indication then the agency may revoke its approval.

After the accelerated approval of Avastin for breast cancer, Genentech, the drug’s sponsor, completed two additional clinical trials and submitted the data from those studies to the FDA, the authors wrote.

The data they retrieved from the additional clinical trials showed only a small effect on tumor growth and no evidence that patients lived a healthier or longer life compared to taking standard chemotherapy alone, which was not enough to outweigh the risk of taking the drug.

When the FDA’s Center for Drug Evaluation and Research, responsible for the approval of Avastin realized that the results of the additional studies on the indication of Avastin did not justify continued approval, they told Genentech that they were proposing to withdraw approval of the indication, but Genentech did not agree with the Center's evaluation of the data and requested a hearing on the Center's withdrawal proposal, with a decision to be made by the Commissioner.

FDA's Oncologic Drugs Advisory Committee (ODAC) voted 6-0 in favor of withdrawing approval of Avastin's breast cancer indication, the authors wrote, and in an earlier meeting of the ODAC, that committee had voted 12-1.

"FDA is committed to working with sponsors to bring promising cancer drugs to market as quickly as possible using tools like accelerated approval," Hamburg said. "I encourage Genentech to consider additional studies to identify if there are select subgroups of women suffering from breast cancer who might benefit from this drug."