FDA approves vismodegib for basal cell cancers

February 1, 2012

The Food and Drug Administration has approved the groundbreaking, first-in-class drug Erivedge (vismodegib, Genentech) to treat adult patients with advanced and metastatic basal cell cancers.

Washington - The Food and Drug Administration has approved the groundbreaking, first-in-class drug Erivedge (vismodegib, Genentech) to treat adult patients with advanced and metastatic basal cell cancers.

Cleared under the FDA’s priority review program - which allows an expedited six-month evaluation of drugs that may offer major treatment advances - Erivedge is intended for use in patients with locally advanced basal cell cancer who are not candidates for surgery or radiation and for patients whose cancer has metastatized.

Erivedge is taken in pill form once a day. It works by inhibiting the Hedgehog pathway, which is active in most basal cell cancers and only a few normal tissues, such as hair follicles, according to the FDA website.

“Our understanding of molecular pathways involved in cancer, such as the Hedgehog pathway, has enabled the development of targeted drugs for specific diseases,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in an FDA press release. “This approach is becoming more common and will potentially allow cancer drugs to be developed more quickly. This is important for patients who will have access to more effective therapies with potentially fewer side effects.”

Erivedge was evaluated in a single, multicenter clinical study in 96 patients with locally advanced or metastatic basal cell carcinoma. The study’s primary endpoint was objective response rate (ORR) or the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment, according to the FDA. Of the patients with metastatic disease receiving Erivedge, 30 percent experienced a partial response and 43 percent of patients with locally advanced disease experienced a complete or partial response.

The most common side effects reported 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.

Erivedge will carry a boxed warning alerting patients and healthcare professionals of the potential risk of death or severe birth effects to a fetus in pregnant women. Pregnancy status must be verified prior to the start of Erivedge treatment.

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