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FDA Approves First Basal Cell Skin Cancer Drug

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The first drug for metastatic basal cell carcinoma (BCC) has been approved by the Food and Drug Administration for adult patients with locally-advanced BCC skin cancer. The drug is also approved for patients whose cancer has recurred following surgery or those who are not candidates for surgery or radiation.

While the drug is promising for cancer patients who once had few options, it can be extremely harmful to unborn children should a woman become pregnant while she or her partner is being treated, the FDA has ruled.

The drug, Erivedge (vismodegib), is a once-a-day capsule that blocks the Hedgehog signaling pathway responsible for proteins that develop into basal cell cancers in adults.

The Hedgehog pathway was first discovered in fruit flies. It was named because mutations in a gene caused a developing fruit fly to look curled up and spiky, resembling a hedgehog. In humans the hedgehog pathway operates at full capacity during normal human embryo development, but becomes inactive in most normal adult tissues.

Basal cell carcinoma, the most common type of skin cancer, is generally a slow growing and painless tumor that starts in the top layer of the skin, called the epidermis. BCC develops on areas of skin that are regularly exposed to sunlight or other ultraviolet radiation.

Richard Pazdur, M.D., director of the FDA’s Center for Drug Evaluation and Research Office of Hematology and Oncology Products, says researchers now have a better understanding of the molecular pathways involved in cancer, such as the Hedgehog pathway. This has enabled the development of targeted drugs for specific diseases.

“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,” Pazdur said.

The drug is marketed by San Francisco-based pharmaceutical company Genentech, part of the Roche group, and was approved nearly six weeks ahead of schedule as part of the FDA’s expedited six-month review process.

The FDA says 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 goal was to determine how many patients, as a percentage, experienced complete and partial shrinkage or disappearance of the cancerous lesions after treatment.

Of the patients with metastatic disease receiving Erivedge, 30 percent experienced a partial response and 43 percent 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, the FDA says.

The FDA approved Erivedge with a “boxed warning” stating the drug should not be taken by women who are pregnant or who could become pregnant. The FDA says the drug has potential risk of death or severe birth effects to an unborn baby and pregnancy status must be verified before starting the treatment.

More important safety information about Erivedge, including reproductive information for women and men, or women whose male partner is taking the drug, is available at: http://www.gene.com/gene/products/information/erivedge/

Lynette Summerill is an award-winning writer and Scuba enthusiast living in San Diego, CA with her husband and two beach loving dogs. In addition to writing about cancer-related issues for EmpowHER, her work has been seen in newspapers and magazines around the world.


FDA Approves Treatment for most common type of skin cancer. FDA News, 30 January 2012. Accessed online 13 February 2012 at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289545.htm

Erivedge Fact Sheet. Genentech. Accessed online 13 February 2012 at: http://www.gene.com/gene/products/information/erivedge/factsheet.html

Reviewed March 1, 2012
by Michele Blacksberg RN
Edited by Jody Smith

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