Scientific Publications by FDA Staff

Clin Cancer Res 2017 May 15;23(10):2377-81

FDA approval summary: sonidegib for locally advanced basal cell carcinoma.

Casey D, Demko S, Shord S, Zhao H, Chen H, He K, Putman A, Helms WS, Keegan P, Pazdur R

On July 24, 2015, the U. S. Food and Drug Administration (FDA) approved sonidegib (ODOMZO(R), Novartis) for treatment of patients with locally advanced basal cell carcinoma (laBCC) not amenable to curative surgery or radiation therapy. The approval was based on data from one randomized, double-blind, non-comparative trial of two doses of sonidegib administered to 230 hedgehog inhibitor-naive patients with metastatic basal cell carcinoma (mBCC, n=36) or laBCC (n=194). Patients were randomized 2:1 to receive sonidegib 800 mg (n=151) or 200 mg (n=79) daily. The objective response rate (ORR) for patients with laBCC was 58% [95% confidence interval (CI): 45, 70] in the 200 mg group and 44% (95% CI: 35, 53) in the 800 mg group. The median duration of response (DoR) for patients with laBCC was non- estimable (NE) in the 200 mg arm and 15.7 months (95% CI: NE) in the 800 mg arm. The ORR for patients with mBCC was 8% (95% CI: 0.2, 36)] and 17% (95% CI: 5, 39)] in patients treated with 200 and 800 mg respectively. The most common AEs occurring in >/=10% of patients were muscle spasms, alopecia, dysgeusia, nausea, fatigue, increased serum creatine kinase (CK), decreased weight, and diarrhea.