On July 18, 2018, the FDA approved 2 new indications for ribociclib (Kisqali; Novartis) for use as (1) initial endocrine-based therapy, in combination with an aromatase inhibitor (AI), for the treatment of pre- and perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer; as well as (2) an initial endocrine-based therapy or after disease progression while receiving endocrine-based therapy, in combination with fulvestrant for postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer.
The approval of ribociclib plus an AI was based on the randomized, double-blind, placebo-controlled MONALEESA-7 study of 495 pre-or perimenopausal women with HR-positive, HER2-negative advanced breast cancer. The estimated median progression-free survival (PFS) was 27.5 months with ribociclib versus 13.8 months with placebo.
The approval of ribociclib plus fulvestrant was based on the MONALEESA-3 study of 726 postmenopausal women. The estimated median PFS was 20.5 months with ribociclib versus 12.8 months with placebo (P <.0001).
The most common (≥20%) adverse reactions with ribociclib in these studies were neutropenia, nausea, infections, fatigue, diarrhea, leukopenia, vomiting, alopecia, headache, constipation, rash, and cough.
These new indications were the first time the FDA used its “Real Time Oncology Review” program and the “Assessment Aid” program, which are pilot programs that enable the FDA to begin analyzing trial data before the drug application is submitted. In this case, this enabled the FDA to approve the drug application <1 month after it was submitted.