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Sonidegib (Odomzo) Gets ‘Thumbs Up’ for Patients With Advanced Basal Cell Carcinoma

Fifty-eight percent of subjects show objective response with 200-mg daily dose

The FDA has given “thumbs up” to sonidegib (Odomzo, Novartis) for the treatment of patients with locally advanced basal cell carcinoma (BCC) that has recurred after surgery or radiation therapy, or who are not candidates for surgery or radiation therapy.

Skin cancer is the most common cancer, and BCC accounts for approximately 80% of non-melanoma skin cancers. BCC starts in 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 BCC 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.

Sonidegib is a once-daily pill. It works by inhibiting the Hedgehog pathway, which is active in BCCs. By suppressing this pathway, Sonidegib may stop or reduce the growth of cancerous lesions.

“Our increasing understanding of molecular pathways involved in cancer has led to approvals of many oncology drugs in difficult-to-treat diseases for which few therapeutic options previously existed,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Thanks to a better understanding of the Hedgehog pathway, the FDA has now approved two drugs for the treatment of basal cell carcinoma in the last three years.” In 2012, vismodegib (Erivedge, Genentech) was the first drug approved to treat locally advanced and metastatic BCC.

The labeling for vismodegib includes a boxed warning alerting health care professionals that the drug may cause death or severe birth defects in a developing fetus when administered to a pregnant woman. A woman’s pregnancy status should be verified before the start of vismodegib treatment, and both male and female patients should be warned about these risks and advised to use effective contraception.

The efficacy of vismodegib was established in a multicenter, double-blind trial, in which 194 patients with locally advanced BCC were randomly assigned to receive either vismodegib 200 mg daily (n = 66) or vismodegib 800 mg daily (n = 128). The study’s primary endpoint was the objective response rate (i.e., the percentage of patients who experienced partial shrinkage or complete disappearance of their tumors). The results showed that 58% of the patients treated with vismodegib 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. The response rates were similar in patients who received vismodegib 800 mg daily; however, adverse effects were more common at that dose.

When administered at a dosage of 200 mg daily, the most common adverse events associated with vismodegib included muscle spasms, alopecia, dysgeusia, fatigue, nausea, musculoskeletal pain, diarrhea, decreased weight, decreased appetite, myalgia, abdominal pain, headache, pain, vomiting, and pruritus. Vismodegib also has the potential to cause serious musculoskeletal-related adverse events, including increased serum creatine kinase levels (with rare reports of rhabdomyolysis), muscle spasms, and myalgia.

Source: FDA; July 24, 2015.

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