National Cancer Institute NCI Cancer Bulletin: A Trusted Source for Cancer Research News
February 7, 2012 • Volume 9 / Number 3

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FDA Update

Drug to Treat Basal Cell Skin Cancer Approved after Priority Review

Last week, the Food and Drug Administration (FDA) approved vismodegib (Erivedge) to treat adults 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 in patients whose cancer has metastasized.

Vismodegib is the first FDA-approved drug for metastatic basal cell carcinoma and was reviewed under the FDA’s priority review program, which provides for an expedited 6-month review of drugs that may offer major advances in treatment.

The drug is a pill taken once a day that inhibits the Hedgehog pathway, a molecular pathway that is active in most basal cell cancers and only a few normal tissues, such as hair follicles.

The safety and effectiveness of vismodegib was evaluated in a single-arm, multicenter clinical study of 104 patients with locally advanced or metastatic basal cell carcinoma. The study’s primary endpoint was objective response rate, which could be evaluated in 96 patients. Thirty percent of the patients with metastatic disease experienced a partial response, and 43 percent of patients with locally advanced disease experienced a complete or partial response.

The most common side effects 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.

Vismodegib was approved with a boxed warning alerting patients and health care professionals of the risk for fetal death or severe birth defects. Pregnancy status must be verified prior to the start of treatment. Patients should be warned about these risks and the need for both male and female patients to use birth control.

Drug Approved for Advanced Kidney Cancer

The Food and Drug Administration (FDA) recently approved axitinib (Inlyta) to treat patients with advanced kidney cancer (renal cell carcinoma) whose disease has not responded to one course of treatment with another drug.

Renal cell carcinoma is a type of kidney cancer that starts in the lining of tubules in the kidney. Axitinib is a pill taken twice a day that works by blocking certain proteins called kinases that play a role in tumor growth and cancer progression.

“This is the seventh drug that has been approved for the treatment of metastatic or advanced kidney cell cancer since 2005,” said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “[These approvals have] significantly altered the treatment paradigm of metastatic kidney cancer, and offer patients multiple treatment options.”

The safety and effectiveness of axitinib were evaluated in a randomized, open-label, multicenter clinical study of 723 patients whose disease had progressed during or after treatment with one prior systemic therapy. Results showed a median progression-free survival of 6.7 months versus 4.7 months with a standard treatment (sorafenib).

The most common side effects observed in the clinical study were diarrhea, high blood pressure, fatigue, decreased appetite, nausea, loss of voice, hand-foot syndrome, weight loss, vomiting, weakness, and constipation. Some patients who took axitinib experienced bleeding, which in some cases was fatal. Patients with untreated high blood pressure, untreated brain metastases, or recent gastrointestinal bleeding should not take axitinib.

Imatinib Granted Expanded Use in Patients with Rare Gastrointestinal Tumors

The Food and Drug Administration (FDA) granted imatinib (Gleevec) regular approval for use in adult patients following surgical removal of CD117-positive gastrointestinal stromal tumors (GIST). CD117, or Kit, is a molecular marker found on most GISTs. The approval highlights an increase in overall survival when the drug is taken for 36 months rather than the standard 12 months.

In a large randomized clinical study, GIST patients who took imatinib for 36 months had significantly longer overall survival, and lived longer without the disease recurring, than those who took imatinib for 12 months. At 60 months, 92 percent of patients who took imatinib for 36 months were alive, compared with 82 percent of patients who took it for 12 months.

Imatinib was originally granted accelerated approval for the treatment of metastatic GIST in 2002. In 2008, imatinib received a subsequent accelerated approval for adjuvant use to treat GIST patients who had had their tumors removed but who were at increased risk for a recurrence. Accelerated approval provides earlier patient access to promising new drugs while confirmatory clinical trials are conducted. Regular approval for the metastatic GIST indication was also granted in 2008.

"The development of [imatinib] over the past decade highlights the need to further study drugs after approval to truly characterize their benefits," said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research. "Although originally approved in the metastatic disease setting, this subsequent trial has demonstrated that longer use of [imatinib] can prolong patient's lives in earlier disease settings."

The most common side effects observed in patients receiving imatinib include swelling (edema), nausea, vomiting, muscle cramps, bone or muscle pain, diarrhea, rash, fatigue, and abdominal pain.

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