FDA approves first cancer drug through new oncology review pilot that enables greater development efficiency
The U.S. Food and Drug Administration today approved Kisqali (ribociclib)
in combination with an aromatase inhibitor for the treatment of
pre/perimenopausal or postmenopausal women with hormone receptor (HR)-positive,
human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic
breast cancer, as initial endocrine-based therapy. The FDA also approved
Kisqali in combination with fulvestrant for the treatment of postmenopausal
women with HR-positive, HER2-negative advanced or metastatic breast cancer, as
initial endocrine based therapy or following disease progression on endocrine
therapy.
This is the first approval that FDA has granted as a part of two new pilot
programs announced earlier this year that collectively aim to make the
development and review of cancer drugs more efficient, while improving FDA’s
rigorous standard for evaluating efficacy and safety. With this real-time
review, the FDA was able to start evaluating the clinical data as soon as the
trial results become available, enabling FDA to be ready to approve the new
indication upon filing of a formal application with the Agency.
The first new program, called Real-Time
Oncology Review, allows for the FDA to review much of the
data earlier, after the clinical trial results become available and the
database is locked, before the information is formally submitted to the FDA.
This allows the FDA to begin its analysis of the data earlier, and provide
feedback to the sponsor on how they can most effectively analyze the data to
answer key regulatory questions. The pilot focuses on early submission of data
that are the most relevant to assessing safety and effectiveness of the
product. Then, when the sponsor submits the application with the FDA, the
review team will already be familiar with the data and in a better position to
conduct a more efficient, timely, and thorough review.
The second program is a new templated Assessment Aid that the
applicant uses to organize its submission into a structured format to
facilitate FDA’s review of the application. By using a structured template, the
FDA is able to layer its assessment into the same file submitted by the
sponsor, allowing this annotated application to serve as the document that
contains the FDA review. This voluntary submission form provides for a more
streamlined approach to reviewing data and illustrating FDA’s analysis. It
allows for drug reviewers to focus on the key benefit-risk and labeling issues
rather than administrative issues.
“With this approval, we’ve demonstrated some of the benefits of the new
programs that we’re piloting for our review of cancer drugs, to improve
regulatory efficiency while enhancing the process for evaluating the data
submitted to us. This shows that, with smart policy approaches, we can gain
efficiency while also improving the rigor of our process. These new programs
were designed to reduce some of the administrative issues that can add to the
time and cost of the review process, including the staffing burdens on the FDA.
For example, by analyzing data earlier in the process, before formal submission
to the FDA, and evaluating submissions in a structured template, we can make it
easier to identify earlier when applications are missing key analysis or
information that can delay reviews,” said FDA Commissioner Scott Gottlieb, M.D.
“With today’s approval, the FDA used these new approaches to allow the review
team to start analyzing data before the actual submission of the application
and help guide the sponsor’s analysis of the top-line data to tease out the
most relevant information. This enabled our approval less than one month after
the June 28 submission date and several months ahead of the goal date.”
These new processes are good for patients, good for health care providers,
good for product developers, and good for the FDA, by allowing our staff to have
more time to engage with product developers and focus on the key aspects of
drug reviews. We can improve efficiency and solidify our gold standard for
review.”
Currently the two pilot programs are being used for supplemental
applications for already-approved cancer drugs and could later be expanded to
original drugs and biologics.
Kisqali was first approved in March 2017 for use with an AI to treat
HR-positive, HER2-negative breast cancer in post-menopausal women whose cancer
is advanced or has spread to other parts of the body.
“The approval adds a new treatment choice for patients with breast
cancer,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of
Excellence and acting director of the Office of Hematology and Oncology
Products in the FDA’s Center for Drug Evaluation and Research. “We are
committed to continuing to bring more treatment options to patients.”
Breast cancer is the most common form of cancer in the United States. The
National Cancer Institute at the National Institutes of Health estimates
approximately 266,120 women will be diagnosed with breast cancer this year and
40,920 will die of the disease. Approximately 72 percent of patients with
breast cancer have tumors that are HR-positive and HER2-negative.
The efficacy of Kisqali in combination with an AI for pre/perimenopausal
women was demonstrated in a clinical trial of 495 participants who received
either Kisqali and an AI or placebo and an AI. All pre- or peri-menopausal
patients on this study received ovarian suppression with goserelin. The trial
measured progression-free survival (PFS), which is generally the amount of time
after the start of this treatment during which the cancer does not
substantially grow and the patient is alive. PFS was longer for patients taking
Kisqali plus an AI (median PFS of 27.5 months) compared to patients who
received placebo plus an AI (median PFS of 13.8 months).
The efficacy of Kisqali in combination with fulvestrant in treating
advanced or metastatic breast cancer was demonstrated in a clinical trial that
included 726 participants who received either Kisqali and fulvestrant or
placebo and fulvestrant. The trial measured PFS, which was longer for patients
taking Kisqali plus fulvestrant (median PFS of 20.5 months) compared to
patients who received placebo plus fulvestrant (median PFS of 12.8 months).
The common side effects of Kisqali are infections, abnormally low count of
a type of white blood cell (neutropenia), a reduction in the number of white
cells in the blood (leukopenia), headache, cough, nausea, fatigue, diarrhea,
vomiting, constipation, hair loss and rash.
Warnings include the risk of a heart problem known as QT prolongation that
can cause an abnormal heartbeat and may lead to death, serious liver problems,
low white blood cell counts that may result in infections that may be severe,
and fetal harm.
The FDA granted Priority Review and Breakthrough
Therapy designation for this indication.
The FDA granted this approval to Novartis Pharmaceuticals Corporation.
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