The FDA has approved the use of Erivedge (vismodegib) to treat locally advanced, metastatic basal cell carcinoma in adult patients who are not candidates for surgery.

The new drug, the first for this indication, is a pill taken once daily, and works by blocking the Hedgehog pathway, which is active in most basal cell cancers and in 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 the FDA's Center for Drug Evaluation and Research, said in a statement. "This approach is becoming more common. This is important for patients who will have access to more effective therapies with potentially fewer side effects."

Erivedge, marketed by Genentech, won approval through the agency's priority review program, which expedites a drug's review process for therapies with the potential to offer major advances in treatment. Basal cell carcinoma is the most common type of skin cancer, and though it is generally curable if diagnosed early, before the cancer has spread, this drug has the potential to cure the advanced, life-threatening cases.

In a multicenter clinical study in 96 locally advanced or metastatic basal cell carcinoma patients, 30 percent had a partial response to the drug and 43 percent had a complete or partial response.

The most common side effects are muscle spasms, hair loss, change in or loss of taste, weight loss, tiredness, nausea, diarrhea, decreased appetite, constipation, vomiting, and joint aches. Other possible side effects are missed monthly periods in females who can become pregnant, low levels of sodium in the blood, low potassium levels, and a higher than normal blood level of urea or other nitrogen-containing compounds in the blood.

The Erivedge label will include a Boxed Warning of the potential risk of death or severe birth effects to a fetus. Pregnancy status should be verified prior to starting treatment, the agency emphasized.