November 18, 2011 — The US Food and Drug Administration (FDA) announced today that it is rescinding its approval of bevacizumab (Avastin, Genentech) for treating metastatic breast cancer because the drug has not proven itself to be safe and effective for that indication.
In a press conference today, FDA Commissioner Margaret Hamburg, MD, explained that bevacizumab received market clearance in 2008 under an accelerated approval pathway that required further studies to confirm its safety and efficacy. Two additional clinical trials, Dr. Hamburg said, failed to confirm the drug's initial promise.
Women who receive bevacizumab for metastatic breast cancer, Dr. Hamburg said, risk potentially fatal adverse events without proof that the drug's use will provide a benefit — specifically, a delay in tumor growth — justifying that risk. In addition, there is no evidence that bevacizumab will help patients live longer or improve their quality of life.
Some of the risks associated with bevacizumab, she said, are severe hypertension; bleeding and hemorrhaging; heart attack or heart failure; and perforations forming in such body parts as the nose, stomach, and intestines.
"Sometimes, despite the hopes of investigators, patients, industry, and even the FDA itself, the results of rigorous testing can be disappointing,"" she said. "This is the case with Avastin when used for metastatic breast cancer."
"I did not come to this decision lightly," Dr. Hamburg said. "As a doctor, woman, and parent, I recognize how frightening and difficult it is to receive a diagnosis of metastatic breast cancer."
Dr. Hamburg noted in today's press conference that the agency's decision does not bar physicians from prescribing bevacizumab on an off-label basis. She advised prudence, however.
"I urge women to discuss the risks and benefits of the drug with their oncologist," she said.
Future reliance on bevacizumab for metastatic breast cancer will depend partly on whether third-party payers are still willing to pay for it. A spokesperson for the Centers for Medicare and Medicaid Services told Medscape Medical News that Medicare "will continue to cover Avastin" and "has no immediate plans to change coverage policies."
"However, CMS will monitor the issue and evaluate coverage options as a result of action by the FDA," the spokesperson said.
Bevacizumab remains an approved treatment for certain kinds of colon, lung, kidney, and brain cancer.
Two Breast Cancer Advocacy Groups Issue Differing Responses
A Genentech spokesperson told Medscape Medical News that the company is not appealing the FDA's decision to revoke approval for bevacizumab for metastatic breast cancer. In a press release, Genentech called the agency action disappointing but said it would nevertheless launch a new phase 3 clinical trial of the drug in combination with paclitaxel in patients with previously untreated metastatic breast cancer and evaluate a potential biomarker that "may help identify which people might derive a more substantial benefit from Avastin."
In today's press conference, Dr. Hamburg said the FDA would welcome a new application from Genentech with data showing that bevacizumab clearly benefits a subset of patients with metastatic breast cancer.
One group that was not disappointed by the FDA's decision is the National Breast Cancer Coalition (NBCC).
"We are gratified that the FDA has continued to support science and put what is best for patients above political, public and industry pressures," the NBCC stated in a press release. "Since 2008, NBCC has taken the position that accelerated approval for Avastin was lowering the bar on drug approval. Based on the results of randomized clinical trials, there is no evidence the drug extends the lives of breast cancer patients; evidence indicates that it does increase the risk of harm.
A response from another breast-cancer advocacy group — Susan G. Komen for the Cure — puts bevacizumab in a more favorable light.
"We want to ensure that women who are successfully using Avastin today continue to have access to the drug, and that their treatment be covered by third-party payers," said Komen president Elizabeth Thompson, who added that her organization respected the length of time that the FDA took to reach its decision.
More information about the FDA's decision is available on the agency's Web site.
Cite this: Avastin No Longer Approved for Breast Cancer, FDA Says - Medscape - Nov 18, 2011.