The US Food and Drug Administration approved Odomzo (sonidegib) for patients with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, or who are not candidates for surgery or radiation therapy.
Odomzo is a pill taken once a day. It works by inhibiting the Hedgehog pathway, which is active in basal cell cancers. By suppressing this pathway, Odomzo may stop or reduce the growth of cancerous lesions.
Odomzo carries a Boxed Warning alerting healthcare professionals that it may cause death or severe birth defects in a developing fetus when administered to a pregnant woman. Pregnancy status should be verified prior to the start of Odomzo treatment, and both male and female patients should be warned about these risks and advised to use effective contraception.
In 2012, Erivedge (vismodegib) was the first drug approved to treat locally advanced and metastatic basal cell carcinoma. Erivedge is marketed by Genentech in San Francisco.
The efficacy of Odomzo was established in a multi-center, double-blind clinical trial, in which 66 patients with locally advanced basal cell carcinoma were randomly assigned to receive Odomzo 200 mg daily and 128 patients were assigned to receive Odomzo 800 mg daily. The study’s primary endpoint was objective response rate, which is the percentage of patients who experienced partial shrinkage or complete disappearance of their tumor(s). Results showed that 58% of patients treated with Odomzo 200 mg had their tumors shrink or disappear. This effect lasted at least 1.9 to 18.6 months, and approximately half of the responding patients’ tumor shrinkage lasted 6 months or longer. Response rates were similar in patients who received Odomzo 800 mg daily. However, side effects were more common at this dose.
At a dose of 200 mg daily, the most common side effects of Odomzo were muscle spasms, alopecia, dysgeusia, fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting, and pruritus. Odomzo also has the potential to cause serious musculoskeletal-related side effects, including increased serum creatine kinase levels with rare reports of rhabdomyolysis, muscle spasms, and myalgia.
Odomzo is marketed by East Hanover, New Jersey-based Novartis Pharmaceuticals Corporation.