biotech drug from Roche has become the first medicine approved to treat
breast cancer before surgery, offering an earlier approach against one of the
deadliest forms of the disease.
The US Food and Drug
Administration, which monitors
food and drug products for safety, approved Perjeta for women with a form of early-stage breast
cancer who face a high risk of having the cancer spreading to other parts of
Surgery to remove tumours
is usually the first step in treating most forms of cancer.
Perjeta is the
first drug to be approved as a pre-surgical step.
Doctors hope that using cancer
drugs earlier could help shrink tumours, making them easier to remove, and in some cases, that could allow women to keep their breasts rather than having a
Doctors also say that treating the disease at its earliest
stages could prevent tumours from returning later, though studies have not yet
established that benefit.
"By making effective
therapies available to high-risk patients in the earliest disease setting, we
may delay or prevent cancer recurrences," said FDA's Dr Richard Pazdur,
who directs the agency's office of cancer products.
Cancer specialists already
use several chemotherapy drugs as initial treatments for cancer, but they are
not formally approved for that particular disease.
The FDA originally approved Perjeta to treat
breast cancer that has spread to other parts of the body after surgery.
The FDA granted the drug
accelerated approval for its new use, based on a study showing women who
received the drug as an initial treatment were more likely to be cancer-free 12
weeks later than women who received older drug combinations.
approval is reserved for drugs that show groundbreaking results for treating
life-threatening diseases in early studies.
As a condition of approval,
Swiss-based Roche's Genentech unit must conduct a larger follow-up study
showing the drug's long-term benefits.
That generally means
showing that patients lived longer or had a higher quality of life due to
taking the drug.
Genentech, located in California, has already enrolled about 4
800 patients in the follow-up study, with results expected in 2016.
Dr Paula Klein, a breast
cancer specialist, says she will start prescribing the drug for early-stage
patients immediately, but stressed the importance of follow-up data on patient
"This does not yet
prove to us that using the antibody in this setting will result in more cures...that is still a hypothesis," said Klein, who directs the breast cancer
programme at Continuum Cancer Centres of New York.
Approval based on study
The FDA said the approval had been based on study of 417 women 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
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 cancerous tumours.
Genentech says this
surgery allowed researchers to confirm the presence or absence of cancer.
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.
A spokeswoman for Genentech
said a regimen of Perjeta plus Herceptin would cost between $27 000 and $49 000,
depending on how long the patient takes the combination. The new Perjeta label
will recommend a course of treatment between nine and 18 weeks.
Facts on Breast cancer
Breast cancer is the second
most deadly form of cancer in US women, behind only lung cancer, 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.
The institute says more than 98% of women who are diagnosed
with first-stage breast cancer survive at least five years.