FDA Approves First US Treatment for Advanced Basal Cell Carcinoma

The US Food and Drug Administration (FDA) has approved the first medicine ever for advanced forms of basal cell carcinoma (BCC), a generally localized skin cancer that in certain rare instances can spread (metastasize) to other parts of the body. The new oral drug, ErivedgeTM (vismodegib), is approved for the treatment of adults whose BCCs have metastasized or recurred after surgery, as well as those whose tumors are deemed untreatable with surgery or radiation.

Close to 2 million Americans each year develop BCC, the most common form of skin cancer.  While it can be disfiguring if not treated early, it generally remains confined to the original tumor site and highly curable. However, in rare cases it can spread to surrounding or distant parts of the body, causing severe deformity (including loss of eyesight or hearing) and potentially life-threatening loss of organ function. These advanced BCCs often may not be effectively treated with surgery or radiation.  Erivedge, developed by Genentech, a member of the Roche Group, helps to shrink these dangerous lesions.

Taken in capsule form once a day, the drug works by acting on the “Hedgehog signaling pathway,” which plays an important role in regulating proper growth and development in early life. It normally becomes inactive in adults, but abnormal Hedgehog signaling is linked to more than 90 percent of BCC cases. Erivedge is designed to inhibit this abnormal signaling in patients with advanced disease.

FDA approval of Erivedge was based on a multicenter Phase II study of 104 patients with advanced BCC. Erivedge shrank lesions in 43 percent of patients with locally advanced BCC and 30 percent of patients with metastatic BCC. Median duration of the response was 7.6 months.

Erivedge is not intended for pregnant women. It can also have side effects ranging from muscle spasms and hair loss to vomiting and diarrhea. Patients are advised to discuss these risks with their physician before taking Erivedge.