The U. S. Food and Drug Administration approved Odomzo® (sonidegib, formerly LDE225) for the treatment of patients with locally advanced basal cell carcinoma (laBCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.
The approval was based on demonstration of a durable objective response rate (ORR) in an international, multi-center, double-blind, randomized, two-arm clinical trial in patients with locally advanced basal cell carcinoma not amenable to local therapy or metastatic basal cell carcinoma (mBCC).
About Basal Cell Carcinoma
BCC consists of abnormal, uncontrolled growths or lesions that arise in the skin’s basal cells, which line the deepest layer of the outermost layer of the skin. BCC accounts for more than 80% of non-melanoma skin cancers. It occurs most frequently on the head and neck, with the nose being the most common site . BCC that spreads from where it started to nearby tissue is called locally advanced disease and can be highly disfiguring . Advanced BCC is thought to represent roughly 1-10% of all cases of BCC . While BCC is generally diagnosed and treated early, it may recur in an estimated 3% of patients after five years . Although BCC rarely becomes advanced, there have been few treatment options at this stage of the disease. Worldwide incidence of BCC is rising by 10% each year due to factors such as an aging population and increased ultraviolet exposure. Incidence rates are estimated to be between 0.003% and 0.55% worldwide.
The clinical trial enrolled 230 patients who were treated 2:1 with either Odomzo® 800 mg (n=151) or 200 mg (n=79) daily until cancer progression or unacceptable side effects. The majority (84%) of those enrolled had laBCC. FDA approval was based on demonstration of durable ORR in these patients with laBCC. The ORR for the 66 patients with laBCC treated with Odomzo® 200 mg arm was 58% consisting of 3 (5%) complete responses and 35 (53%) partial responses.
A similar response rate was noted in the 128 patients with laBCC treated with Odomzo® 800 mg. Among the 38 responding patients with laBCC in the 200 mg arm, 7 patients (18%) experienced subsequent disease progression, and 4 of these 7 patients had maintained a response of 6 months or longer. The remaining 31 patients (82%) continue to respond with ongoing responses ranging from 1.9+ to 18.6+ months; 16 patients have ongoing responses of 6 months or longer, and the median duration of response has not been reached.
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 National Cancer Institute. NCI Dictionary of Cancer Terms “locally advanced cancer.” Available at: http://www.cancer.gov/publications/dictionaries/
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