Sonidegib: A Review in Locally Advanced Basal Cell Carcinoma

Abstract

Sonidegib (Odomzo®), an oral smoothened (SMO) antagonist, is indicated for the treatment of adults with locally advanced basal cell carcinoma (laBCC) who are not candidates for surgery or radiation therapy, or adults with recurrent laBCC following surgery or radiation therapy. In the multicentre BOLT trial, the primary endpoint (i.e., an objective tumour response rate point estimate of ≥30 % and a 95 % confidence interval lower bound of >20 % in patients with fully assessable laBCC and all patients with metastatic BCC) was met at the primary analysis cut-off date (median follow-up 13.9 months) in the sonidegib 200 mg (36 % [95 % CI 24–50]) and 800 mg (34 % [95 % CI 25–43]) once-daily groups. Sonidegib 200 mg once daily (recommended dosage) had a better benefit-risk profile than the 800 mg dosage. Central review of the patients with laBCC in this population showed that 43 % achieved an objective response with sonidegib 200 mg once daily at the primary analysis date. Clinically meaningful responses were sustained in the sonidegib 200 mg group, based on an 18-month analysis. The majority of treatment-emergent adverse events were of mild to moderate severity and manageable with dosage adjustments, concomitant medications and/or non-drug therapies (e.g., adequate hydration). The acceptable benefit-risk profile of sonidegib, along with a paucity of treatment options and the seriousness of the condition, makes sonidegib an emerging option for the treatment of adults with laBCC that has recurred following surgery or radiation therapy, or in those who are not candidates for surgery or radiation therapy