FDA Approval Summary: Sonidegib for Locally Advanced Basal Cell Carcinoma

Abstract On July 24, 2015, the FDA approved sonidegib (ODOMZO; Novartis) for the treatment of patients with locally advanced basal cell carcinoma (laBCC) not amenable to curative surgery or radiotherapy. The approval was based on data from one randomized, double-blind, noncomparative trial of two doses of sonidegib administered to 230 hedgehog inhibitor–naïve 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 for patients with laBCC was nonestimable (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 adverse events occurring in ≥10% of patients were muscle spasms, alopecia, dysgeusia, nausea, fatigue, increased serum creatine kinase, decreased weight, and diarrhea. Clin Cancer Res; 23(10); 2377–81. ©2017 AACR.