Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
Clin Cancer Res. 2017 May 15;23(10):2377-2381. doi: 10.1158/1078-0432.CCR-16-2051. Epub 2017 Jan 10.
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, = 36) or laBCC ( = 194). Patients were randomized 2:1 to receive sonidegib 800 mg ( = 151) or 200 mg ( = 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. .
2015 年 7 月 24 日，FDA 批准 sonidegib（ODOMZO；诺华公司）用于治疗无法进行根治性手术或放疗的局部晚期基底细胞癌（laBCC）患者。该批准基于一项针对 230 例无 hedgehog 抑制剂治疗史的转移性基底细胞癌（mBCC，n = 36）或局部晚期基底细胞癌（laBCC，n = 194）患者的随机、双盲、非对照试验数据。患者按 2:1 的比例随机分为 sonidegib 800 mg 组（n = 151）和 200 mg 组（n = 79），每日接受治疗。laBCC 患者的客观缓解率（ORR）在 200 mg 组为 58%（95%CI，45-70），800 mg 组为 44%（95%CI，35-53）。laBCC 患者的中位缓解持续时间在 200 mg 组无法评估（NE），800 mg 组为 15.7 个月（95%CI，NE）。mBCC 患者的 ORR 在 200 mg 组和 800 mg 组分别为 8%（95%CI，0.2-36）和 17%（95%CI，5-39）。发生≥10%患者的最常见不良反应是肌肉痉挛、脱发、味觉障碍、恶心、疲劳、血清肌酸激酶升高、体重下降和腹泻。
