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FDA Approves Kisqali for Advanced Breast Cancer, Thanks to Innovative New Process


Thanks to two pilot programs, the FDA was able to more efficiently approve Kisqali for certain patients with advanced breast cancer.

The Food and Drug Administration (FDA) approved the combination regimen of Kisqali (ribociclib) plus an aromatase inhibitor for the treatment of pre/perimenopausal or postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer. The treatment will be used as the first endocrine-based therapy for these patients.

Kisqali was also approved to be used in combination with fulvestrant to treat postmenopausal women who have HR-positive, HER2-negative advanced or metastatic breast cancer, as the initial endocrine therapy after their disease progresses.

The former approval was based on a clinical trial which proved that Kisqali plus and aromatase inhibitor had better progression-free survival (PFS) outcomes compared to patients who received placebo plus an aromatase inhibitor (27.5 months versus 13.8 months, respectively).

The latter approval (Kisqali plus fulvestrant) was based on a clinical trial including more than 720 patients. PFS for the combination was 20.5 months compared with 12.8 months in the fulvestrant plus placebo arm.

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 severe infections and fetal harm.

This is not only a breakthrough for the patients who can receive the treatment, but also for the FDA, as this is the first approval granted as a part of two pilot programs: the Real-Time Oncology Review and the Assessment Aid.

The Real-Time Oncology Review allows the FDA to review information from clinical trials before it is officially submitted to the organization. In doing so, it allows the FDA to communicate with the sponsors so that they can continue their research in an effective manner to answer important regulatory questions.

On the other hand, the Assessment Aid helps an applicant put their FDA submission into an organized and structured format that will help the FDA review it in an efficient manner.

“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,” said FDA Commissioner Scott Gottlieb, M.D., in a statement.

Currently, the two pilot programs will only be used for drugs that are already FDA-approved and are submitting applications for additional indications. Kisqali fits this bill, as it was previously approved in March 2017 to be used with an aromatase inhibitor to treat HR-positive, HER2-negative metastatic breast cancer.

“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, in a statement. “We are committed to continuing to bring more treatment options to patients.”

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