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FDA approves first drug for metastatic basal cell carcinoma
The FDA today approved vismodegib for the treatment of adults with basal cell carcinoma.
The FDA approval, granted under the agency’s priority review program, allows vismodegib (Erivedge, Genentech) to be used by adults with locally advanced basal cell carcinoma who cannot be treated with radiation or surgery, or those whose disease has spread to other parts of the body.
Vismodegib is the first FDA-approved drug for metastatic basal cell carcinoma, the most common form of skin cancer. Vismodegib — taken once a day — inhibits the Hedgehog pathway, which is active in most basal cell cancers.
“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 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 FDA — which was not expected to announce its decision on the drug until March 8 — issued its approval based on results of a single, multicenter clinical study of 96 patients with locally advanced or metastatic basal cell carcinoma. The primary endpoint was objective response, defined as complete or partial shrinkage or disappearance of cancerous lesions after treatment.
Thirty percent of patients with metastatic disease who received vismodegib experienced partial response, and 43% of patients with locally advanced disease experienced complete or partial response, according to the FDA. The most common adverse effects reported in patients who received vismodegib included muscle spasms, hair loss, weight loss, nausea, diarrhea and fatigue.
The approval comes with a boxed warning urging health care professionals to verify patients’ pregnancy status before treatment. The warning will indicate that vismodegib can increase the risk for death or other severe effects for a fetus.
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Basal cell carcinoma is the most common malignancy, with an estimated 1 million cases per year. Most are localized and easily treated with local therapies such as surgical excision. However, occasionally these tumors can become locally advanced, making excision difficult with the risk of severe disfigurement. Even more uncommon is the development of distant metastatic disease. Therefore, locally advanced or metastatic basal cell carcinoma is a relatively uncommon problem of a very common disease. Treatment options for this group of patients have been severely lacking.
Up to this point, there has not been a medical therapy that has demonstrated benefit in this setting. Vismodegib, a first-in-class small molecule inhibitor of the hedgehog pathway, has — for the first time in a large clinical trial — demonstrated clinically significant responses in both locally advanced and metastatic basal cell carcinoma patients. Now there is a medical alternative to disfiguring surgery or large-field irradiation for patients with extensive, locally advanced basal cell carcinoma. This medication is likely to significantly change the standard of care for this group of patients.
Karl D. Lewis, MD
Assistant professor of medicine
Associate director of the Melanoma Research Clinics
University of Colorado Denver
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