FDA approves vismodegib for treating basal cell skin cancer

FDA has approved vismodegib (Erivedge, Curis and Genentech, a member of the Roche Group) for the treatment of adult patients with basal cell carcinoma, the most common type of skin cancer. It is the first FDA-approved drug for metastatic basal cell carcinoma.

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 spread to other parts of the body (metastatic).

Erivedge was reviewed under 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 being approved ahead of the March 8, 2012, prescription user-fee goal date.

Basal cell carcinoma is generally a slow growing and painless form of skin cancer that starts in the top layer of the skin (epidermis). The cancer develops on areas of skin that are regularly exposed to sunlight or other ultraviolet radiation.

Erivedge is a tablet taken once a day and works by inhibiting the Hedgehog pathway, a pathway that is active in most basal cell cancers and only a few normal tissues, such as hair follicles.

"Our understanding of molecular pathways involved in cancer, such as the Hedgehog pathway, has enabled the development of targeted drugs for specific diseases," Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research, said 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."

The safety and effectiveness of Erivedge was evaluated in a single, multicenter clinical study in 96 patients with locally advanced or metastatic basal cell carcinoma. The clinical study's primary end point was objective response rate or the percentage of patients who experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment. Of the patients with metastatic disease receiving the drug, 30% experienced a partial response and 43% of patients with locally advanced disease experienced a complete or partial response.

The most common side effects observed in patients treated with the drug 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 is being approved with a Boxed Warning alerting patients and healthcare professionals of the potential risk of severe birth effects or death to a fetus. Pregnancy status must be verified prior to the start of treatment. Male and female patients should be warned about these risks and the need for birth control.