The Food and Drug Administration has approved ribociclib (Kisqali) in combination with an aromatase inhibitor (AI) 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.
Ribociclib was first approved in March 2017 for use with an AI to treat HR-positive, HER2-negative advanced breast cancer in postmenopausal women.
Approval for ribociclib in combination with an AI for pre/perimenopausal women was based on progression-free survival (PFS) in MONALEESA-7, a trial of premenopausal women with HR-positive, HER2-negative, advanced breast cancer. The women received either ribociclib and an AI, or placebo and an AI, and all also received ovarian suppression with goserelin (Zoladex). Of 495 women who received nonsteroidal AIs, median PFS was 27.5 months for women also receiving ribociclib, versus 13.8 months for women who received placebo plus the AI.
Approval for ribociclib in combination with fulvestrant in treating advanced or metastatic breast cancer was based on PFS results from MONALEESA-3, which enrolled 726 women with HR-positive, HER2-negative, advanced breast cancer who received no or up to one line of prior endocrine therapy. Median PFS was 20.5 months for women randomized to receive ribociclib and fulvestrant, compared with 12.8 months for women randomized to receive placebo plus fulvestrant.
The common side effects of ribociclib are infections, neutropenia, leukopenia, headache, cough, nausea, fatigue, diarrhea, vomiting, constipation, hair loss, and rash. Warnings include the risk of QT prolongation, serious liver problems, low white blood cell counts, and fetal harm, the FDA said.
This is the first FDA approval as part of two new pilot programs announced earlier this year: Real-Time Oncology Review allows for the FDA to review much of the data earlier, before the information is formally submitted to the FDA, and the Assessment Aid is a structured template that offers a more streamlined approach.
“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 the press statement. “This enabled our approval less than 1 month after the June 28 submission date and several months ahead of the goal date.”
The two pilot programs are currently being used for supplemental applications for already approved cancer drugs and could later be expanded to original drugs and biologics, the FDA said.
Ribociclib is marketed as Kisqali by Novartis Pharmaceuticals Corporation.