FDA Expands Use of Ribociclib in Breast Cancer

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The FDA has expanded its approval of ribociclib (Kisqali) to treat hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced or metastatic breast cancer under 2 new pilot programs to make the development and review of cancer drugs more efficient.

The FDA approved ribociclib (Kisqali) for 2 indications. The first indication is in combination with an aromatase inhibitor as initial endocrine-based therapy for pre/perimenopausal or postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer. The second indication is in combination with fulvestrant as an initial endocrine-based therapy or after disease progression on endocrine therapy for the treatment of postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer.

The approval of Kisqali also represents the first under 2 new pilot programs: Real-Time Oncology Review and Assessment Aid.

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


Real-Time Oncology Review allows the FDA to review much of the data after the clinical trial results become available and before the information is formally submitted to the FDA. The pilot focuses on early submission of data that are most relevant to assessing the safety and efficacy of a product. Under Assessment Aid, the applicant submits in a structure format to facilitate the FDA’s review of the application.

The programs are currently only being used for supplemental applications for already-approved cancer drugs, but their use may be expanded to original products.

Kisqali is already approved to be used with an aromatase inhibitor 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.

"Compelling data for Kisqali have led to the broadest first-line indications of any CDK4/6 inhibitor," Liz Barrett, CEO of Novartis Oncology, said in a statement. "With this new approval Kisqali has the potential to help even more people in the US live a longer life without progression of disease from this incurable form of breast cancer."

The expanded approval was based on a clinical trial of 495 patients who received either Kisqali with an aromatase inhibitor or a placebo with an aromatase inhibitor. The median progression-free survival (PFS) for patients on the Kisqali regimen was 27.5 months compared with 13.8 months for patients taking placebo.

A trial of 726 patients found that Kisqali taken with fulvestrant also improved PFS over placebo. The median PFS for patients on the Kisqali regimen was 20.5 months compared with 12.8 months for patients taking placebo with fulvestrant.

“The approval adds a new treatment choice for patients with breast cancer,” said Richard Pazdur, MD, 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.”