Regulatory Focus™ > News Articles > FDA Approves Drug for Rare Brain Tumor in Kids

FDA Approves Drug for Rare Brain Tumor in Kids

Posted 30 August 2012

The U.S. Food and Drug Administration (FDA) approved a new pediatric dosage of a drug used to treat a rare brain tumor, according to an agency release.

Afinitor Disperz is the first drug to be approved for children affected by inoperable subependymal giant cell astrocytoma (SEGA). The approval allows patients ages 1 or older with tuberous sclerosis complex (TSC) to use the drug treatment.

"Today's approval demonstrates the value of further studying a drug to better characterize its benefits and how it should be used in pediatric patients," Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research said in the release.

FDA's release said TSC is a rare genetic disease causing 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.

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