Drug to Treat Basal Cell Skin Cancer Approved after Priority Review ► NCI Cancer Bulletin for February 7, 2012 - National Cancer Institute

NCI Cancer Bulletin for February 7, 2012 - National Cancer Institute

Drug to Treat Basal Cell Skin Cancer Approved after Priority Review

Last week, the Food and Drug Administration (FDA) approved vismodegib (Erivedge) to treat adults with basal cell carcinoma, the most common type of skin cancer. The drug is intended for use in patients with locally advanced basal cell cancer who are not candidates for surgery or radiation and in patients whose cancer has metastasized.
Vismodegib is the first FDA-approved drug for metastatic basal cell carcinoma and was reviewed under the FDA’s priority review program, which provides for an expedited 6-month review of drugs that may offer major advances in treatment.
The drug is a pill taken once a day that inhibits the Hedgehog pathway, a molecular pathway that is active in most basal cell cancers and only a few normal tissues, such as hair follicles.
The safety and effectiveness of vismodegib was evaluated in a single-arm, multicenter clinical study of 104 patients with locally advanced or metastatic basal cell carcinoma. The study’s primary endpoint was objective response rate, which could be evaluated in 96 patients. Thirty percent of the patients with metastatic disease experienced a partial response, and 43 percent of patients with locally advanced disease experienced a complete or partial response.
The most common side effects were muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted sense of taste, decreased appetite, constipation, vomiting, and loss of taste function in the tongue.
Vismodegib was approved with a boxed warning alerting patients and health care professionals of the risk for fetal death or severe birth defects. Pregnancy status must be verified prior to the start of treatment. Patients should be warned about these risks and the need for both male and female patients to use birth control.