lundi 3 septembre 2012

FDA approves first drug formulated for children with rare brain tumor

The U.S. Food and Drug Administration recently approved Afinitor Disperz (everolimus tablets for oral suspension), a new pediatric dosage form of the anti-cancer drug Afinitor (everolimus) used to treat a rare brain tumor called subependymal giant cell astrocytoma (SEGA). Afinitor Disperz is the first approved pediatric-specific dosage form developed for the treatment of a pediatric tumor.

Afinitor Disperz is recommended to treat patients ages 1 year and older with tuberous sclerosis complex (TSC) who are diagnosed with SEGA that cannot be treated with surgery. Prior to approval of this new dosage form, Afinitor was recommended for use only in patients ages 3 years old and older. Afinitor was granted accelerated approval in 2010 to treat SEGA in patients with TSC.

TSC is a rare genetic disease that causes tumors to grow in the brain and other vital organs. SEGA is a slow-growing tumor that can cause life-threatening complications by blocking the flow of fluid in the brain. It is considered a major diagnostic feature of TSC and is seen in 6 percent to 9 percent of patients, generally pediatric and young adult patients.

Everolimus, the active ingredient in Afinitor and Afinitor Disperz, blocks the uncontrolled activity of a protein called the mTOR kinase, which plays a critical role in the development and growth of SEGA tumors occurring in patients with TSC.

The FDA has previously approved Afinitor to treat adults with advanced renal cell carcinoma that has progressed after treatment with other cancer therapies (2009); adults with progressive advanced neuroendocrine tumors of pancreatic origin (2011); adults with TSC who have renal angiomyolipomas not requiring immediate surgery (2012); and for use in combination with Aromasin (exemestane) to treat certain postmenopausal women with advanced hormone-receptor positive, HER2-negative breast cancer (2012).

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