August 29, 2012
1 min read

FDA approves pediatric dosage of everolimus for subependymal giant cell astrocytoma

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The FDA today approved a pediatric dosage form of everolimus to treat subependymal giant cell astrocytoma.

It is the first approved pediatric-specific dosage form developed for the treatment of a pediatric tumor.

Everolimus (Afinitor Disperz, Novartis) is recommended to treat patients aged 1 year and older with tuberous sclerosis complex who are diagnosed with inoperable subependymal giant cell astrocytoma (SEGA). Prior to approval of the pediatric dosage, everolimus was recommended for use only in patients aged 3 years and older.

The FDA granted everolimus accelerated approval in 2010 to treat SEGA in patients with tuberous sclerosis complex.

“Appropriate pediatric dosage forms, such as Afinitor Disperz, help to ensure the safe and effective use of oncology drugs in children,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, said in a press release. “In addition, 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.”

Available in smaller dose increments than its adult dosage counterpart, the pediatric dosage of everolimus dissolves easily in a small volume of water, allowing for easier administration to patients who are unable to swallow whole tablets.

The FDA based its approval on updated data from a single-arm, 28-patient study that was used to support the drug’s accelerated approval in 2010.

The agency also evaluated results of a more recent study of 117 pediatric and adult patients who were randomly assigned to receive either everolimus or a placebo daily. Study results demonstrated that 35% of patients treated with everolimus exhibited tumor shrinkage, compared with none who were treated with placebo.

Everolimus should be used only in patients with tuberous sclerosis complex who require treatment for SEGA that cannot be surgically removed. The most commonly observed adverse effects in patients with SEGA were mouth ulcers and respiratory tract infections.

Both adult and pediatric dosages of everolimus remain under accelerated approval for the treatment of SEGA in patients with tuberous sclerosis complex. Studies are under way to further evaluate the long-term safety and effectiveness of everolimus in pediatric and adult patients with SEGA.