* FDA holds rare two-day hearing
* Patients say Avastin helps, data shows little benefit
* FDA new drugs director says cannot "evergreen" approval
* Roche shares down slightly
(Adds company statement)
By Anna Yukhananov
SILVER SPRING, Md., June 28 Breast cancer
patients testified that Roche Holding AG's ROG.VX Avastin
saved their lives as U.S. health officials weighed whether the
world's best-selling cancer drug should remain approved for
At stake in the hearing that began Tuesday is nearly $1
billion in annual Avastin revenues, the hopes of cancer
patients and the resolve of the Food and Drug Administration to
stick to data-based decisions.
"I am alive due to Avastin," said 66-year-old Patricia
Howard. "I'm a wife, mother, sister, aunt and granny. I'm not
just a statistic. It's in your hands to make sure I don't
Patient testimony began the unusual two-day meeting to hear
Roche's appeal of an FDA proposal in December to withdraw
Avastin's approval for breast cancer treatment.
At least 40 patients held signs and chanted outside the FDA
building on Tuesday, wearing pink T-shirts with the slogan "We
are statistically significant."
But FDA presenters said the agency had to follow the data.
"We cannot permit sponsors to evergreen approval of a drug
that has not been shown to be safe and effective," Dr. John
Jenkins, director of the FDA's Office of New Drugs, told the
FDA officials said the agency could review approval of
Avastin for breast cancer after new studies, but it would be
inappropriate to keep the breast cancer approval as the studies
would take at least three years.
For Switzerland-based Roche, the FDA decision could shave
almost a billion dollars off Avastin's more than $6 billion in
annual sales. Roche shares fell 0.4 percent in European trading
Roche unit Genentech, which will present its case on
Wednesday, said it agreed with the many women and their doctors
who testified on Tuesday. "Women with this incurable disease
should have the option to choose this medicine," it said in a
Some patients said Genentech should offer free Avastin for
those patients that respond well to the drug while it studies
which specific patient populations could benefit.
For patients, an FDA rejection of the appeal could mean
insurance companies would stop covering the expensive drug for
breast cancer, potentially jeopardizing treatment for an
estimated 17,000 women currently using the medicine.
Breast cancer is the second-leading type of cancer among
women after skin cancer, with 1 in 8 women in the United States
expected to develop invasive breast cancer in their lifetime.
In 2010, an estimated 200,000 new cases of invasive breast
cancer were diagnosed in the United States, and 40,000 women
died from the disease.
The panel will make its recommendation at the end of the
hearing on Wednesday, and FDA Commissioner Margaret Hamburg
will later make the final decision.
LATER STUDIES LESS PROMISING
Avastin won U.S. clearance for breast cancer in 2008 based
on a study showing the drug stalled 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 to confirm the drug worked. [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.
Genentech has been fighting the decision, saying 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.
Some medical experts argue that if the FDA keeps the breast
cancer indication solely to placate patient groups, it would
undermine the integrity of the accelerated approval process.
"If the FDA demonstrates that it is unable or unwilling to
withdraw accelerated approval when the totality of evidence
fails to meet its standard for regular approval, such a
precedent risks undermining ... the agency's credibility as it
seeks to regulate medical products for the public good," said a
perspective article published in the New England Journal of
Medicine on Monday.
Stripping the breast cancer indication for Avastin would
not prevent doctors from using it. Without FDA approval,
insurers may refuse to pay Avastin's $8,000-a-month price tag,
and Genentech would not be able to promote Avastin for that
Most analysts believe the panel will not change its mind,
especially as five of the six members voted against Avastin's
use in breast cancer at a hearing last July.
Medicare, the U.S. federal health insurer, is likely to cut
coverage for Avastin in breast cancer if the FDA revokes that
use. That could shrink U.S. sales by half in 2011 to $400
million, said Karen Andersen, analyst at Morningstar.
But she sees a 60 percent chance that private health
insurers would still keep coverage for Avastin on an off-label
(Reporting by Anna Yukhananov; Editing by Michele Gershberg
and Tim Dobbyn)