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August 02, 2015 – The American Food and Drug Administration (FDA) very recently approved Sonidegib (Odomzo) to treat 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.
Sonidegib (Odomzo) works by inhibiting a molecular pathway, called the Hedgehog pathway, which is active in basal cell cancers. By suppressing this pathway, Sonidegib (Odomzo) may stop or reduce the growth of cancerous lesions. In 2012, Vismodegib (Erivedge) was the first drug approved to
treat locally advanced and metastatic basal cell carcinoma. Thanks to an ever increasing understanding of the Hedgehog pathway and its involvement in the etiology of basal cell carcinoma, the FDA has been able to approve two drugs for the treatment of basal cell carcinoma just in the last three years.
The efficacy of Sonidegib (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 Sonidegib (Odomzo) 200 mg daily and 128 patients were assigned to receive Sonidegib (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 percent of patients treated with Sonidegib (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 six months or longer. Response rates were similar in patients who received Sonidegib (Odomzo) 800 mg daily, however side effects were more common at this dose.
Sonidegib (Odomzo) comes with a selection of serious adverse effects. Thus, at a dose of 200 mg daily, the most common side effects of Sonidegib (Odomzo) were muscle spasms, alopecia (hair loss), dysgeusia (distortion in the sense of taste), fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia (muscle pain), abdominal pain, headache, pain, vomiting and pruritus (itching). Sonidegib (Odomzo) also has the potential to cause serious musculoskeletal-related side effects, including increased serum creatine kinase levels [with rare reports of muscle tissue breakdown (rhabdomyolysis)], muscle spasms, and myalgia.
Sonidegib (Odomzo) also carries a Boxed Warning alerting healthcare professionals that Sonidegib (Odomzo) 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 Sonidegib (Odomzo) treatment, and both male and female patients should be warned about these risks and advised to use effective contraception.
Skin cancer is an important indication. It is the most common cancer and basal cell carcinoma accounts for approximately 80 percent of non-melanoma skin cancers. Basal cell carcinoma starts in the top layer of the skin (called the epidermis) and usually develops in areas that have been regularly exposed to the sun and other forms of ultraviolet radiation. According to the National Cancer Institute, the number of new cases of non-melanoma skin cancer appears to be increasing every year. Locally advanced basal cell skin cancer refers to basal cancers that have not spread to other parts of the body, but cannot be curatively treated with local treatments, specifically surgery and radiation.