First Drug Formulated for Children with Rare Brain Tumor Approved

The most common side effects observed in patients with SEGA were mouth ulcers and respiratory tract infections.

Afinitor Disperz is recommended to treat patients ages 1 year and older with tuberous sclerosis complex (TSC) who are diagnosed with SEGA that cannot be treated with surgery. Prior to approval of this new dosage form, Afinitor was recommended for use only in patients ages 3 years old and older. Afinitor was granted accelerated approval in 2010 to treat SEGA in patients with TSC.

Appropriate pediatric dosage forms, such as Afinitor Disperz, help to ensure the safe and effective use of oncology drugs in children, said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDAs Center for Drug Evaluation and Research. In addition, todays approval demonstrates the value of further studying a drug to better characterize its benefits and how it should be used in pediatric patients.

Afinitor Disperz is available in smaller dose increments than the adult dosage form, Afinitor. Afinitor Disperz also dissolves easily in a small volume of water, making it easy to administer to patients who are unable to swallow whole tablets to take their medication.

Afinitors manufacturer, Novartis, also provided updated safety and efficacy data from the single-arm study of 28 pediatric and adult patients used to support the drugs accelerated approval in 2010 for the treatment of SEGA in patients with TSC. The company also supplied new information from a more recent study of 117 pediatric and adult patients who were randomly assigned to take Afinitor or a placebo daily. Results showed 35 percent of patients treated with Afinitor experienced tumor shrinkage, compared with none who were treated with placebo.

Afinitor Disperz should be used only in patients with TSC who require treatment for SEGA that cannot be surgically removed. The most common side effects observed in patients with SEGA were mouth ulcers and respiratory tract infections.

TSC is a rare genetic disease that causes tumors to grow in the brain and other vital organs. SEGA is a slow-growing tumor that can cause life-threatening complications by blocking the flow of fluid in the brain. It is considered a major diagnostic feature of TSC and is seen in 6 percent to 9 percent of patients, generally pediatric and young adult patients.

Everolimus, the active ingredient in Afinitor and Afinitor Disperz, blocks the uncontrolled activity of a protein called the mTOR kinase, which plays a critical role in the development and growth of SEGA tumors occurring in patients with TSC.

Afinitor and Afinitor Disperz remain under accelerated approval for the treatment of SEGA in patients with TSC. Studies are ongoing to further evaluate the long-term safety and effectiveness of Afinitor and Afinitor Disperz in pediatric and adult patients with SEGA. Afinitor Disperz is classified as an orphan drug because it is intended to treat a rare disease or condition. Afinitor Disperzs application was granted priority review, which FDA completed in six months.

The FDA has previously approved Afinitor to treat adults with advanced renal cell carcinoma that has progressed after treatment with other cancer therapies (2009); adults with progressive advanced neuroendocrine tumors of pancreatic origin (2011); adults with TSC who have renal angiomyolipomas not requiring immediate surgery (2012); and for use in combination with Aromasin (exemestane) to treat certain postmenopausal women with advanced hormone-receptor positive, HER2-negative breast cancer (2012).

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First Drug Formulated for Children with Rare Brain Tumor Approved