A U.S. panel dealt a blow to Roche Holding AG’s multibillion-dollar cancer drug Avastin on Tuesday
, urging U.S. officials to revoke the medicine’s approval for breast cancer after concluding studies showed insufficient benefit for patients.
If regulators follow that advice, the Swiss drugmaker could no longer promote Avastin for that use in the United States.
Doctors still could prescribe Avastin for breast cancer as it would retain approval for colon, lung, brain and kidney cancers, but sales likely would fall.
Breast cancer treatment accounts for about $1 billion of Avastin’s more than $6 billion in annual sales, analysts said. The product is Roche’s top-selling drug, accounting for 13 percent of sales in 2009.
Members of a Food and Drug Administration panel said they were not convinced Avastin provided worthwhile benefits in advanced breast cancer. The drug did not extend patients’ lives but delayed cancer growth by up to three months.
That improvement was not enough to justify serious complications, panelists said. They voted 12-1 to urge the FDA to remove the breast cancer approval.
The drug’s risks include gastrointestinal perforations, bleeding and blood clots.
Roche said rates of those problems were low, at less than 4 percent in the breast cancer trials.
But panel chairman Wyndham Wilson, a National Cancer Institute researcher, said there was definitive evidence that Avastin causes serious and life-threatening side effects. “Small numbers, but if you’re the one, that’s not what you want to be exposed to,” he said.
Overall data “does not support this being effective” in advanced breast cancer, Wilson added.
The FDA usually follows panel recommendations. A decision is due by September 17.
The vote was a rare setback for Avastin, a widely used treatment for a variety of cancers. The drug has been forecast to be the world’s biggest selling prescription medicine by 2014, with annual sales topping $9 billion a year.
Morningstar analyst Karen Andersen said the negative ruling in breast cancer “wouldn’t be something that would change my valuation” of Roche.
“Colorectal and lung cancer are both markets where Avastin has been able to penetrate a very high percentage of the first-line patients. (Roche) really relies on those indications for the foundation of Avastin sales,” she said.
Withdrawal of the breast cancer approval would curb, but not eliminate, sales for that use, Sanford Bernstein analyst Tim Anderson said.
“Even with savage and pessimistic assumptions, perhaps $1 billion of 2015 sales could go,” or about a 5 percent hit to earnings per share, Anderson said in a research note.
Roche unit Genentech said the company stands by its data showing Avastin helps patients with advanced breast cancer. “Avastin should be an option for patients with this incurable disease,” a company statement said.
The committee action “does not change the current availability of Avastin for women with advanced breast cancer,” Genentech added.
Avastin won clearance for breast cancer in 2008 under a shorter approval process, but Roche was required to run two follow-up studies to confirm the drug’s effectiveness and receive full approval.
Those studies failed to confirm the level of benefit seen in the initial breast cancer trial, FDA reviewers said.
Avastin, which is given intravenously, delayed the growth of cancer by 5.5 months in the initial study. In the two later studies, the time ranged from about one month to three months.
Avastin, which works by starving tumors of the blood vessels they need to grow, did not extend overall survival in any of the studies. Advanced breast cancer patients generally live about 18 to 24 months, the FDA said.
Roche said the extension of time without cancer growing was meaningful for patients with advanced breast cancer. At that stage the disease cannot be cured and the goal is to stabilize patients as much as possible.
The company also said the rate of treatment-related deaths in breast cancer trials was the same for Avastin or comparison groups, at 1.8 percent.
Roche argued for expansion of Avastin’s approved breast cancer uses to include combining the drug with other types of chemotherapy, but the committee also rejected those proposals.
A panel that met in December 2007 voted 5-4 against Avastin’s initial approval for breast cancer, but the FDA approved that use in 2008 based on benefits seen in the earlier breast cancer trial. The drug’s generic name is bevacizumab.
Earlier this month, Britain’s health cost watchdog said it was rejecting Avastin for breast cancer due to “uncertain evidence.”
The drug’s wholesale cost is about $8,000 per month for advanced breast cancer. Roche limits the annual cost for any FDA-approved use to about $57,000.
The FDA is not allowed to consider cost when deciding on drug approvals.