FDA Approves Kisqali for Use in Pre/Perimenopausal Women with Breast Cancer

This is the first approval that the FDA has granted as part of its new approaches to streamlining the development and review of oncology drugs.

Officials with the FDA have approved ribociclib (Kisqali, Novartis) in combination with an aromatase inhibitor (AI) for the treatment of pre/perimenopausal or postmenopausal women with advanced or metastatic breast cancer, according to an agency press release.

The combination is indicated to treat 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 ribociclib plus 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.

The approval was granted as part of 2 new pilot programs launched by the FDA earlier this year to facilitate the development and review of oncology drugs while also improving the FDA’s standard for evaluating efficacy and safety. The programs allowed the agency to begin evaluating clinical data as soon as trial results became available, enabling a quicker path to approval.

Ribociclib 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 metastatic.

The newly-approved indication was based on data from a clinical trial of 495 participants. Patients received either ribociclib with an AI or a placebo with an AI. All pre- or peri-menopausal women in this study received ovarian suppression with goserelin.

According to the trial results, progression-free survival (PFS) was longer for patients taking ribociclib plus an AI (median PFS of 27.5 months) compared with patients who received placebo plus an AI (median PFS of 13.8 months).

“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,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “This enabled our approval less than 1 month after the June 28 submission date and several months ahead of the goal date.”

The 2 pilot programs, the Real-Time Oncology Review and templated Assessment Aid, include approaches such as the early submission of clinical data to facilitate analysis and a structured submission template to enable a more streamlined strategy for reviewing data.

The FDA granted Priority Review and Breakthrough Therapy designation for this indication.


FDA approves first cancer drug through new oncology review pilot that enables greater development efficiency [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613801.htm?utm_campaign=07182018_PR_FDA%20expands%20the%20use%20of%20breast%20cancer%20drug&utm_medium=email&utm_source=Eloqua. Accessed July 18, 2018.