FDA Approves Everolimus (Afinitor Disperz) for Rare Pediatric Brain Tumor

August 31, 2012

The FDA approved a dissolvable form of everolimus (Afinitor Disperz) for the treatment of children with subependymal giant cell astrocytoma (SEGA) that cannot be treated with surgery.

The US Food and Drug Administration (FDA) approved everolimus tablets for oral suspension (Afinitor Disperz) earlier this week. The drug, a new pediatric dosage form of everolimus (Afinitor), is used to treat patients with subependymal giant cell astrocytoma (SEGA), a rare brain tumor, that cannot be treated with surgery. This dissolvable form of everolimus represents the first approved  drug developed for the treatment of a pediatric tumor with a pediatric-specific dosage.

The new drug is indicated to treat patients who are 1 year or older with tuberous sclerosis complex (TSC) who are diagnosed with SEGA that cannot be surgically removed and is classified as an orphan drug due to the rarity of the condition.

While the mTOR inhibitor everolimus has already been approved for a number of indications, this new pediatric form dissolves easily in a small volume of water, making it easy to administer to young patients, and it is available in smaller dose increments than the standard form.

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

TSC is a rare genetic disease that causes tumors to grow in the brain and other vital organs. SEGA-seen in 6% to 9% of patients with TSC-is a benign, slow-growing tumor that usually forms in the walls of fluid-filled spaces in the brain and can cause life-threatening complications. The tumors-which are made up of large, star-shaped cells called astrocytes-are typically seen in pediatric and young adult patients.

Safety and efficacy data that led to the approval were from a single-arm study of 28 pediatric and adult patients used to support the drug’s accelerated approval in 2010 for the treatment of SEGA in patients with TSC (that indication was for patients older than 3). A more recent study randomized 117 pediatric and adult patients to either daily everolimus or placebo-35% of patients in the experimental arm experienced tumor shrinkage, compared with none in the placebo arm.The most common adverse events observed in patients with SEGA were mouth ulcers and respiratory tract infections.

Everolimus has previously been approved by the FDA for the treatment of RCC (2009), advanced pancreatic neuroendocrine tumors (2011), and in combination with exemestane (Aromasin) to treat certain breast cancers (2012).