FDA approves first pediatric dosage for rare brain tumor

Novartis (NYSE: NVS) has been granted approval for the first pediatric-specific dosage to be used to treat a pediatric tumor by the U.S. Food and Drug Administration. A new formulation of its anti-cancer drug Afinitor will be used to treat a rare brain tumor called subependymal giant cell astrocytoma or SEGA in children aged one and older for children with tuberous sclerosis complex, according to a statement from the FDA.

The orphan drug is for patients with these conditions for whom surgery is not an option. Although Afinitor Disperz had been granted accelerated approval in 2010 to treat SEGA in patients with tuberous sclerosis, it was only available to treat children aged 3 years old and up.

Tuberous sclerosis complex is a rare, genetic condition that generates benign tumors that grow on major organs like the brain, kidneys and skin, and SEGA occurs in about 6 percent to 9 percent of the patient population. The benign, slow growing brain tumor blocks off the flow of fluid to the brain.

Tuberous sclerosis complex is estimated to occur in one in 6,000 births and nearly 1 million people worldwide have the condition, according to the Tuberous Sclerosis Complex Alliance.

Some analysts project Novartis’ sales of Afinitor will grow to $2.2 billion by 2017. Among the other indications for the drug are renal cancer and a rare form of pancreatic cancer. Most recently, it was approved by European regulators to treat breast cancer. The Swiss drug maker’s U.S. headquarters are in East Hanover, New Jersey.

 

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