National Cancer Institute NCI Cancer Bulletin: A Trusted Source for Cancer Research News
April 7, 2009 • Volume 6 / Number 7

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FDA Update

New Treatment for Advanced Kidney Cancer Approved

The FDA has approved everolimus (Afinitor) for advanced renal cell carcinoma in patients whose disease has progressed after treatment with one of two other targeted therapies, sunitinib (Sutent) and sorafenib (Nexavar).

Everolimus (Afinitor) logo

The approval is based on the most recent results of a 416-patient, phase III trial dubbed RECORD-1—presented last fall at the European Society for Medical Oncology annual meeting—which showed that everolimus treatment improved progression-free survival by 67 percent compared with patients given placebo, 4.9 months versus 1.9 months.

Everolimus targets a protein known as mTOR, which affects tumor cell division, angiogenesis, and cell metabolism. According to the drug's manufacturer, Novartis, everolimus is being investigated as a treatment for other cancers in which mTOR is thought to play a prominent role, including breast, gastric, and liver cancer, and non-Hodgkin lymphoma. NCI's Cancer Therapy Evaluation Program is sponsoring the development of studies that will include the use of everolimus to treat neuroendocrine tumors.

ODAC Recommends Approval of Bevacizumab for GBM

Last week, the FDA's Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of accelerated approval of bevacizumab (Avastin) for previously treated glioblastoma multiforme (GBM). Accelerated approval is granted to treatments for life-threatening diseases on the basis of preliminary evidence, with the requirement that additional studies be conducted to confirm a true clinical benefit.

Bevacizumab (Avastin) logo

The drug's manufacturer, Genentech, requested accelerated approval based on results from two early phase clinical trials, neither of which had a control arm. The first trial included 85 patients from several study sites, and approximately one quarter had partial responses, or tumor shrinkage of greater than 50 percent. There were no complete responses, meaning none of the tumors completely disappeared. One-year survival was nearly 38 percent, Genentech noted in its presentation to the committee, compared with the 25 percent historically seen in patients treated with salvage chemotherapy. In the second trial, a single-center, 56-patient study led by Dr. Howard Fine of NCI's Center for Cancer Research (CCR) at the NIH Clinical Center, the objective response rate was approximately 20 percent, with no complete responses.

FDA staff argued in a briefing document that using MRI to assess the effect of bevacizumab on GBM tumors is complicated because the drug affects the shape and function of blood vessels, making it difficult to determine whether it shrank the tumor or simply reduced swelling around the tumor. However, even a reduction in swelling, known as edema, can have important quality of life implications for patients, committee members noted, in addition to any tumor-related effects.

"What influenced the committee, in looking at the totality of data, is the overall sense from the two trials that there really was a benefit to patients associated with bevacizumab," said ODAC member and CCR Senior Investigator Dr. Wyndham Wilson.

The FDA is expected to make a decision about approval by May, according to Genentech, and a global, randomized phase III trial of bevacizumab in patients with newly diagnosed GBM will start some time in 2009.

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