FDA approves smaller-dose Afinitor for rare brain tumor in children

SILVER SPRING, Md. — The Food and Drug Administration has approved a new formulation of a cancer drug made by Novartis for a type of brain tumor in children, the agency said.

The FDA announced the approval of Afinitor Disperz (everolimus) tablets for oral suspension to treat a rare brain tumor called subependymal giant-cell astrocytoma, or SEGA. The agency said Afinitor Disperz, a new dosage form of the drug Afinitor, is the first pediatric-specific dosage form for a pediatric tumor to receive FDA approval.

The drug, designed to dissolve in water, is recommended for patients aged 1 year and older with tuberous sclerosis complex who are diagnosed with SEGA that can't be treated with surgery. TSC is a rare genetic disease that causes tumors to grow in the brain and other vital organs, while SEGA is a slow-growing tumor that can cause life-threatening complications by blocking the flow of blood to the brain, occurring in 6-9% of TSC patients.


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