FDA approves new treatment for most common type of skin cancer
On January 30, the U.S. Food and Drug Administration (FDA) announced that it had approved Erivedge (vismodegib) for the treatment of adult patients with basal cell carcinoma, the most common type of skin cancer.
The drug 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 metastasized to other parts of the body. The drug, which was developed by the U.S. unit of Roche Holding AG,will be marketed in the U.S. by Genentech Inc. Many new drugs come with a hefty price tag: a monthly supply of once-a-day capsules will cost $7,500.
Erivedge was reviewed under the FDA’s priority review program; it is the first drug approved by the agency for metastatic basal cell carcinoma. The priority review program provides for an expedited six-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 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,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “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, multi-center clinical study in 96 patients with locally advanced or metastatic basal cell carcinoma. The clinical 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. Of the patients with metastatic disease receiving Erivedge, 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 Erivedge 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 death or severe birth effects to a fetus (unborn baby). Pregnancy status must be verified prior to the start of Erivedge treatment. Male and female patients should be warned about these risks and the need for birth control.