drug from Roche moved one step closer Thursday to becoming the first
medicine approved to treat breast
cancer before surgery.
The US Food and Drug
Administration's panel of cancer experts voted 13-0, with one abstention, that
the benefits of Perjeta as an initial treatment for breast cancer outweigh its
risks. The recommendation is not binding, but sets the stage for the FDA to
clear the drug as the first pharmaceutical option approved to shrink or eliminate
A study by Swiss drug maker
Roche Holding AG's Genentech unit showed women who received Perjeta as initial
treatment were 18% more likely to be cancer-free after 12 weeks than women who
received older drug combinations.
Perjeta is already approved
to treat breast cancer that has spread to other parts of the body, known as
metastatic cancer. But Genentech is seeking approval to market the drug as the
first step in treating the disease.
Panel chairman Dr Mikkael
Sekeres called the vote "a historic moment".
"We are supporting the
movement of a highly active drug for metastatic breast cancer to the first-line
setting, with the hope that women with earlier stages of breast cancer will
live longer and better," said Sekeres, an associate professor of medicine
at the Cleveland Clinic.
Doctors hope that using
cancer drugs earlier could help shrink tumours, making them easier to remove.
In some cases, that could allow women to keep their breasts, rather than having
a full mastectomy. Cancer specialists already use several chemotherapy drugs as
initial treatments for cancer, but they are not formally approved for the use.
The FDA, which monitors the
safety of drugs and food products, is considering granting Perjeta accelerated
approval, a step used to speed up the introduction of drugs that have shown
groundbreaking results in early testing.
But panellists stressed
that Genentech must conduct more trials to prove that Perjeta's early promise
ultimately results in longer life expectancy for patients.
"I look forward to the
day several years from now when we can say that this improves survival,"
said Dr Louis Diehl of Duke University Medical Centre. "But if it
doesn't, I think we should stand up and say we did the very best we could
today, but it didn't work out."
Genentech, based in South
San Francisco, said it is has already enrolled nearly 4800 patients in a large
study to confirm Perjeta's effectiveness.
The panel based its vote on
a 417-woman study comparing Perjeta in different combinations against older
breast cancer treatments. When Perjeta was combined with Herceptin, another
Genentech drug, and standard chemotherapy, 39% of women saw their cancer
reach undetectable levels. Only 21% of women experienced the same
results from taking Herceptin and chemotherapy alone. After drug treatment, all
the women received standard breast surgery to remove any tumours. Genentech
says this surgery allowed researchers to confirm the presence or absence of
While panellists ultimately
backed the drug's benefits, they pointed to a number of shortcomings with the
trial, including its small population size and its experimental measure of
effectiveness. Genentech measured patients' initial response to the drug, but
it's not yet clear whether those patients will ultimately live longer,
The study also showed
evidence of worrisome side effects, including a higher rate of heart problems among
patients taking Perjeta.
FDA leadership acknowledged
these issues, but urged the panel to consider the potential advantages of
getting the drug to market quickly.
"The other side of the
equation is you have to look at the benefit, and that is the introduction of an
agent that may help many women prevent metastatic disease," said Dr
Richard Pazdur, director of the FDA's office of cancer drugs.
Meeting the criteria
Earlier in the week, FDA
scientists published a very positive review of Perjeta, indicating that it met
their criteria for accelerated approval. The FDA is scheduled to make a
decision on the drug by 31 October.
The panel also heard from
breast cancer survivors who emphasized the importance of starting treatment as
early as possible.
"Reducing the overall
time battling the disease has an immense and dramatic effect on the financial,
emotional and medical well-being of our lives," said Judy Hodges of
Richmond, Virginia, who was diagnosed with breast cancer in 2006. Hodges spoke
as a volunteer for the National Patient Advocate Foundation, a non-profit group
founded by cancer patients.
Like Herceptin, Perjeta
only works in a subset of about 20% of breast cancer patients who have tumours
that overproduce a protein known as HER-2. This protein causes cancer cells to
divide and grow faster than usual.
Breast cancer is the second
most deadly form of cancer in US women, and is expected to kill more than
39,000 Americans this year, according to the National Cancer Institute. About
6000 to 8000 deaths per year are attributed to the HER-2 form of the disease.
Breast cancer is highly
treatable when detected early. More than 98% of women who are diagnosed with
first-stage breast cancer survive at least five years, according to NCI