FDA approves new
treatment for most common type of skin cancer
Washington, D.C. On Jan. 30, 2012, Erivedge (vismodegib) was approved by the
U.S. Food and Drug Administration to treat adult patients with basal cell
carcinoma, the most common type of skin cancer. The Erivedge 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 spread to other
parts of the body (metastatic).
Erivedge, reviewed under the agency’s priority review program, is
the first FDA-approved drug for metastatic basal cell carcinoma. Erivedge
was reviewed under the FDA’s priority review program that 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 is a pill taken once a day and 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 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.
Erivedge is being approved with a BOXED WARNING alerting patients
and health care 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.
Erivedge is marketed by South San Francisco based-Genentech, a member of the
For more information:
FDA Voice Blog: Skin
Deep: New Skin Cancer Therapies Offer Progress for Patients
FDA: Office of Hematology
and Oncology Products
FDA: Approved Drugs:
Questions and Answers
The FDA, an agency within the U.S.
Department of Health and Human Services, protects the public health by
assuring the safety, effectiveness, and security of human and veterinary
drugs, vaccines and other biological products for human use, and medical
devices. The agency also is responsible for the safety and security of our
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