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FDA Approval Summary: Sonidegib for Locally Advanced Basal Cell Carcinoma
Denise Casey1, Suzanne Demko2, Stacy Shord2
1Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland. denise.casey@fda.hhs.gov.
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.
Insights
Sonidegib (ODOMZO) is FDA-approved for locally advanced basal cell carcinoma (laBCC). Clinical trials show varying objective response rates for sonidegib in laBCC and metastatic basal cell carcinoma (mBCC) patients.
Area of Science:
- Oncology
- Dermatology
- Pharmacology
Background:
- Basal cell carcinoma (BCC) is the most common human cancer.
- Locally advanced basal cell carcinoma (laBCC) and metastatic basal cell carcinoma (mBCC) present treatment challenges.
- Hedgehog pathway inhibitors represent a therapeutic advancement for advanced BCC.
Purpose of the Study:
- To evaluate the efficacy and safety of sonidegib (ODOMZO) for treating patients with laBCC.
- To compare two different doses of sonidegib in hedgehog inhibitor-naïve patients.
- To assess sonidegib's impact on objective response rate (ORR) and duration of response (DoR) in advanced BCC.
Main Methods:
- Randomized, double-blind, noncomparative trial involving 230 patients with laBCC or mBCC.
- Patients received either 200 mg or 800 mg of sonidegib daily.
- Objective response rate (ORR) and duration of response (DoR) were key efficacy endpoints.
Main Results:
- For laBCC patients, the ORR was 58% with 200 mg sonidegib and 44% with 800 mg.
- Median DoR was nonestimable (NE) in the 200 mg arm and 15.7 months in the 800 mg arm for laBCC.
- For mBCC patients, ORRs were 8% (200 mg) and 17% (800 mg).
- Common adverse events included muscle spasms, alopecia, and dysgeusia.
Conclusions:
- Sonidegib (ODOMZO) demonstrated efficacy in treating patients with laBCC.
- The 200 mg dose showed a higher ORR in laBCC patients compared to the 800 mg dose.
- Sonidegib is a viable treatment option for advanced BCC, with a manageable safety profile.
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