* FDA advisers vote 6-0 risks outweigh benefits
* Patients say drug keeps them alive
* Roche stands to lose $1 billion
* Roche says will still conduct follow-up study (Adds American Cancer Society comment)
By Anna Yukhananov and Alina Selyukh
SILVER SPRING, Md., June 29 U.S. health advisers unanimously rejected use of the drug Avastin for breast cancer, dealing a blow to its manufacturer and patients who insisted that the medicine saved their lives.
Advisers to the U.S. Food and Drug Administration agreed that the Roche Holding AG ROG.VX drug was not safe or clinically beneficial, based on several years of follow-up trials.
Their vote endorsed an FDA move in December to revoke its approval for Avastin in breast cancer. FDA Commissioner Margaret Hamburg is due to make a final decision later.
"No matter what way we look at it, there's nothing we can hang our hat on in these studies that would make me feel comfortable continuing to expose a lot of patients to risk without a clear benefit," said Mikkael Sekeres, associate professor of medicine at the Cleveland Clinic Taussig Cancer Institute and one of the panel members.
Patient advocates broke down in tears after the vote in an emotional conclusion to the rare two-day hearing. Without FDA approval, insurers and government health programs likely will stop covering the $8,000-a-month drug, potentially jeopardizing treatment for an estimated 17,000 women using the medicine.
"I'm on Avastin alone, so it's not the chemo that's keeping me alive," said Christi Turnage, 49, who stood up among a handful of breast cancer patients after the vote, shouting at the panel members and crying.
Turnage said she has been taking Avastin for eight years, but that her insurance plan had warned her they would drop coverage if it is rejected by the FDA. She cannot afford it on her own.
"Trial or no trial, we have nothing. I don't even qualify for a single (drug) trial in the United States or Canada because I have no symptoms of disease," she said.
The American Cancer Society said there were conflicting opinions on the drug, and some insurers would continue to cover Avastin.
"The FDA clearly believes there is no evidence of significant benefit in metastatic breast cancer," said Dr. Leonard Lichtenfeld, deputy chief medical officer for the American Cancer Society. "But there are other highly-qualified experts ... who are clearly on the other side of that assessment. ... We end up in a situation where we have dueling science."
He said political backlash against the FDA's move is sure to continue.
IMPACT ON SALES
Roche stands to lose nearly $1 billion out of a total of $6 billion in Avastin revenue if its use in breast cancer is ultimately revoked, though that outcome has already been assumed by its investors.
"Avastin has sales in breast cancer in other markets and for many other indications, (so) this really does not have a significant impact on our evaluation of Roche overall," said Karen Andersen, an analyst at Morningstar.
She estimates that Avastin's U.S. breast cancer sales would fall from $800 million in 2010 to around $400 million in 2011.
Roche's Genentech unit sought more time for Avastin, asking that it remain available to breast cancer patients while the company conducts more studies on its clinical benefits.
FDA officials said the side effects and the fact it would take at least three and a half years to get new trial results meant they could not continue to back its use in the interim.
"The agency has to look at protecting a larger number of patients," said Dr. Ralph Freedman, panel member and clinical professor of gynecologic oncology at the University of Texas. "Sometimes they have to make a decision that doesn't favor individual patients on the basis of the whole."
Breast cancer is the second-most common type of cancer among women after skin cancer. One in eight U.S. women are expected to develop invasive breast cancer during their lives.
Avastin won U.S. clearance for the disease in 2008 based on a study showing the drug stalled breast cancer growth by 5.5 months more when used in combination with standard chemotherapy. As part of an accelerated approval, the FDA required Roche to run follow-up studies. [ID:nN1E75P03C]
Later studies found only a one- to three-month delay in breast cancer growth. None of the studies showed Avastin extended the lives of patients with advanced breast cancer.
Some patients also had severe side effects, including holes in the stomach and intestines, severe bleeding and blood clots. The company says the incidence of these serious side effects was just under 3 percent greater for those taking Avastin.
In December, the FDA proposed revoking Avastin's clearance for breast cancer, while keeping the drug on the market for colon, lung, brain and kidney cancers.
"We are very disappointed by the committee's recommendation and hope the Commissioner does not decide to remove an important medicine for women with this incurable disease who already have too few treatment options," said Dr. Hal Barron, chief medical officer at Genentech.
The company said it would still conduct follow-up studies to confirm Avastin's efficacy, regardless of the FDA's decision. They will look to see whether Avastin works best with a specific type of chemotherapy called paclitaxel and try to identify genetic markets that could pinpoint which patients would be respond to treatment. (Additional reporting by Toni Clarke and Deena Beasley; Editing by Michele Gershberg and Robert MacMillan)