(Repeats June 26 story for additional readers with no changes
to headline or text)
* What: FDA advisory panel to vote on Avastin
* When: Two-day hearing starts on Tuesday
* Lack of new evidence makes approval unlikely-analysts
* Almost $1 billion in Avastin sales at stake
* Private insurance may still pay, not Medicare-analyst
By Anna Yukhananov
WASHINGTON, June 26 (Reuters) -- Drugmaker Roche Holding AG
ROG.VX will try to convince U.S. regulators to reverse course
on Avastin, the world's best-selling cancer medicine, and
approve its use for breast cancer in a hearing this week.
But analysts say the Food and Drug Administration, which
proposed removing the breast cancer indication in December, is
unlikely to change its opinion without new evidence about
Avastin's ability to help breast cancer patients live longer.
"There seems to be only a very slim chance that Avastin
will stay on the market for breast cancer," said Tim Race,
analyst at Deutsche Bank in London. "It would be a major U-turn
for the FDA ... and there would be quite a lot of fallout from
such a change of stance."
For Swiss-based Roche, a FDA decision could shave off
almost a billion dollars in annual Avastin sales, according to
And for patients, an FDA rejection of the appeal could mean
insurance companies would stop covering the expensive drug,
potentially jeopardizing treatment for an estimated 17,000
women currently using the medicine, advocates say.
After skin cancer, breast cancer is the second-leading type
of cancer among women, according to the Centers for Disease
SURVIVAL VERSUS QUALITY OF LIFE
Avastin won U.S. clearance for breast cancer in 2008 based
on a study showing the drug stalled cancer growth by 5.5
months. As part of an accelerated approval, the FDA required
Roche to run follow-up studies to confirm the drug worked. For
timeline, see [ID:nN1E75M15P]
Later studies found only a one- to three-month delay in
breast cancer growth. And no studies showed Avastin extended
the lives of patients with advanced breast cancer.
The drug also had side effects, including holes in the
stomach and intestines, severe bleeding and blood clots.
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. For details, see
Genentech, a Roche unit, 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.
"We looked at metastatic cancer, where almost everyone will
have a recurrence of their disease," said Dr. Philippe Bishop,
head of global clinical development for Avastin. "We look for
how long a patient can live without their disease getting
Roche also claimed the rate of side effects was low, less
than 4 percent in breast cancer trials.
Stripping the breast cancer indication for Avastin would
not prevent doctors from using it. But 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
Many breast cancer groups and U.S. lawmakers were outraged
by the FDA's December decision on Avastin, charging it was
based on cost, something the FDA is forbidden from doing. They
also accused the agency of trying to limit patient options and
dictate medical care.
In a rare move, the FDA granted Genentech an appeal,
allowing the company to make its case at a public hearing on
Tuesday and Wednesday. It is the first time the agency has had
an appeal hearing over its decision to withdraw the breast
cancer indication under accelerated approval, the FDA said.
Dr. Stanley Waintraub, a chief of breast oncology at the
John Theurer Cancer Center at Hackensack University Medical
Center in New Jersey, plans to testify in favor of Avastin.
He said 17,000 women in the United States are currently
using the medicine, including some of his patients.
"I have been a doctor since 1977, and I have been in
oncology since 1982," he said. "I have watched too many
patients die. This drug works. It doesn't work all the time.
But nothing works all the time, not even an aspirin.
"If the FDA opposes this drug, it would be a horrible day
in history for patients in this country."
FOCUS ON GENETIC MARKERS
Most analysts believe the panel will not change its mind,
especially as five of the six panelists voted against Avastin's
use in breast cancer at a hearing last July.
This time around, panelists will also have an option to
keep Avastin's indication for breast cancer, pending further
Roche studies that show a more significant clinical benefit.
"They do have an opportunity to say that there's not a lot
of harm that can be done while we wait (to see) what happens
with that additional trial," said Ira Loss, an analyst with
Washington Analysis. He thinks it's possible the breast cancer
use will not be removed completely.
Roche has also said it plans to run studies that could
pinpoint genetic markers to identify who would best respond to
Avastin. The FDA has said it is open to any new research that
can identify specific types of women likely to benefit.
"Roche has been searching for years for the right
biomarker. At this point, it would be an unexpected outcome if
the FDA decided to preserve the label while they're waiting for
that data," said Morningstar analyst Karen Andersen.
IMPACT ON SALES
Analysts estimate about $1 billion of Avastin's more than
$6 billion in yearly sales come from breast cancer uses. The
product used to be Roche's top-selling drug, and some expected
it to become the world's biggest-selling drug by 2014.
But its prospects have dimmed after doubts about its
benefits in breast cancer and after it failed in clinical
trials for prostate and stomach cancer.
Morningstar's Andersen said her projections for Roche's
overall earnings would not be greatly affected by the FDA's
decision. Avastin is still used for breast cancer in other
countries, and European authorities relaxed curbs on the drug
in April after initially restricting use. [ID:nLDE73E12N]
An FDA revocation would likely see Medicare, the U.S.
federal health insurer, cut coverage for the drug, shrinking
U.S. sales by half in 2011 to $400 million, Andersen said.
She sees a 60 percent chance that private health insurers
would still maintain coverage for Avastin on an off-label
(Reporting by Anna Yukhananov, additional reporting by Ben
Hirschler in London; editing by Michele Gershberg and Jeffrey