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FDA approves pediatric dosage of everolimus

The US Food and Drug Administration (FDA) has approved a new pediatric dosage form of everolimus for the treatment of subependymal giant cell astrocytoma (SEGA), a rare brain tumor in children. The everolimus tablet for oral suspension is the first approved pediatric-specific dosage form developed for the treatment of a pediatric tumor.

The new formulation is recommended to treat patients aged 1 year and older with tuberous sclerosis complex (TSC) who are diagnosed with SEGA that cannot be treated with surgery. Before approval of this new dosage form, everolimus was recommended for use only in patients aged 3 years and older.

Tuberous sclerosis complex is a rare genetic disease that causes tumors to develop in the brain and other vital organs. Subependymal giant cell astrocytoma, found in 6% to 9% of patients with TSC, is a slow-growing tumor that can cause life-threatening complications by impeding the flow of fluid in the brain. Everolimus blocks the uncontrolled activity of the protein mTOR kinase that affects the development and growth of SEGA tumors.

Everolimus tablets for oral suspension are available in smaller dose increments than the adult dosage form. The new formulation also dissolves easily in a small volume of water, which is beneficial for patients who are unable to swallow whole tablets.

The new drug is classified as an orphan drug. The application was granted priority review; FDA completed the review in 6 months. Approval was based on a single-arm study of 28 pediatric and adult patients. In a more recent study of 117 pediatric and adult patients, 35% of those treated with everolimus and none of those who received placebo showed tumor shrinkage.

The most common adverse effects of everolimus seen in patients with SEGA are mouth ulcers and respiratory tract infections.

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