• Anand Shah, Erik Bloomquist, Shenghui Tang, Wentao Fu, Youwei Bi, Qi Liu, Jingyu Yu, Ping Zhao, Todd R Palmby, Kirsten B Goldberg, C J George Chang, Paresma Patel, Elleni Alebachew, Amy Tilley, William F Pierce, Amna Ibrahim, Gideon M Blumenthal, Rajeshwari Sridhara, Julia A Beaver, and Richard Pazdur.
• Center for Drug Evaluation and Research, U.S. Food and Drug, Administration, Silver Spring, Maryland. anand.shah@fda.hhs.gov.
• Clin Cancer Res. 2018 Jul 1; 24 (13): 2999-3004.

AbstractOn March 13, 2017, the FDA approved ribociclib (KISQALI; Novartis Pharmaceuticals Corp.), a cyclin-dependent kinase 4/6 inhibitor, in combination with an aromatase inhibitor as initial endocrine-based therapy for the treatment of postmenopausal women with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer. The approval was based on a randomized, double-blind, placebo-controlled, international clinical trial (MONALEESA-2). A total of 668 patients were randomized to receive either ribociclib plus letrozole (n = 334) or placebo plus letrozole (n = 334). An improvement in progression-free survival (PFS) was observed in patients receiving ribociclib plus letrozole compared with patients receiving placebo plus letrozole [HR = 0.556; 95% confidence interval (CI), 0.429-0.720]. Overall response rate (ORR) in patients with measurable disease was 52.7% (95% CI, 46.6-58.9) in the ribociclib plus letrozole arm and 37.1% (95% CI, 31.1-43.2) in the placebo plus letrozole arm. Overall survival data were immature. The most common adverse reactions observed in 20% or more of patients taking ribociclib were neutropenia, nausea, fatigue, diarrhea, leukopenia, alopecia, vomiting, constipation, headache, and back pain. This article summarizes FDA decision-making and data supporting the approval of ribociclib. Clin Cancer Res; 24(13); 2999-3004. ©2018 AACRSee related commentary by Spring and Bardia, p. 2981.©2018 American Association for Cancer Research.

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