title: 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.
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland. denise.casey@fda.hhs.gov.
authors.forename: Denise
authors.initials: D
authors.lastname: Casey
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Suzanne
authors.initials: S
authors.lastname: Demko
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Stacy
authors.initials: S
authors.lastname: Shord
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Hong
authors.initials: H
authors.lastname: Zhao
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Huanyu
authors.initials: H
authors.lastname: Chen
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Kun
authors.initials: K
authors.lastname: He
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Alexander
authors.initials: A
authors.lastname: Putman
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Whitney
authors.initials: W
authors.lastname: Helms
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Patricia
authors.initials: P
authors.lastname: Keegan
authors.affiliation: Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
authors.forename: Richard
authors.initials: R
authors.lastname: Pazdur
mesh_terms: D001713:Biphenyl Compounds / Q000008:administration & dosage* / Q000009:adverse effects; D002280:Basal Cell Carcinoma / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D018572:Disease-Free Survival; D004311:Double-Blind Method; D017277:Drug Approval; D064420:Drug-Related Side Effects and Adverse Reactions / Q000473:pathology; D005260:Female; D053823:Hedgehog Proteins / Q000235:genetics; D006801:Humans; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000473:pathology; D011725:Pyridines / Q000008:administration & dosage* / Q000009:adverse effects; D015398:Signal Transduction / Q000187:drug effects; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D001713:Biphenyl Compounds; D053823:Hedgehog Proteins; D011725:Pyridines; C561435:sonidegib