9/11/2012
New indication for AFINITOR Disperz Illustrates the Value of Considering Adult Therapies for Pediatric Applications
National Brain Tumor Society, the largest nonprofit organization dedicated to the brain tumor community in the United States, is pleased with the Food and Drug Administration’s (FDA) recent approval for a pediatric dosage of AFINITOR® Disperz (everolimus tablets for oral suspension) as a therapy for subependymal giant cell astrocytoma (SEGA), a brain tumor seen with a genetic condition called tuberous sclerosis (TS).[1]
With only a few brain tumor therapies reaching FDA approval during the last two decades, the National Brain Tumor Society encourages the industry to aggressively evaluate pediatric applications of other (currently approved) adult therapies, and provide them to patients as soon as they are determined to be safe and effective. In addition, National Brain Tumor Society is especially gratified to see the FDA exercising using the accelerated approval process to advance a new treatment option for a tumor type disproportionately impacting children.
According to Paul Graham Fisher M.D, Chief, Division of Child Neurology, Stanford University and Lucile Packard Children’s Hospital, “We hope that the FDA and other federal agencies in collaboration with industry will build off this approval timeline (adult accelerated approval in 2010 and pediatric dosage in 2012) to help shorten the amount of time it takes for adult therapies to be evaluated for pediatric treatment effect in clinical settings and ultimately be approved for safe and effective treatment of more pediatric brain tumor types.”
Key Facts:
- Brain Tumors affect approximately 688,000 Americans
- An estimated 138,000 are malignant (cancerous) and 550,000 benign
- About 7% of brain tumors occur in children age 19 and younger
- There are about 120 types of brain tumors
About subependymal giant cell astrocytoma (SEGA) is “a benign (not cancerous), slow-growing tumor that usually forms in the walls of fluid-filled spaces in the brain. The tumors are made up of large, star-shaped cells called astrocytes. Subependymal giant cell astrocytomas are common in patients with tuberous sclerosis (an inherited disorder in which benign tumors form in the brain and other parts of the body).” [2] Tuberous sclerosis--also called tuberous sclerosis complex (TSC)1--is a rare, multi-system genetic disease that causes benign tumors to grow in the brain and on other vital organs such as the kidneys, heart, eyes, lungs, and skin. It usually affects the central nervous system and results in a combination of symptoms including seizures, developmental delay, behavioral problems, skin abnormalities, and kidney disease. [3]
SEGA occurs in approximately 6 percent to 9 percent of patients, generally pediatric and young adult patients. [4] Approximately 1 in 6,000 children have tuberous sclerosis and about 50,000 in the United States. [5]
Sources:
[1] According to FDA information, 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. See http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm317385.htm
[2] National Cancer Institute, http://www.cancer.gov/dictionary?cdrid=689357
[3] National Institute for Neurological Disorders and Stroke, http://www.ninds.nih.gov/disorders/tuberous_sclerosis/detail_tuberous_sclerosis.htm
[4] Food and Drug Administration, http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm317385.htm
[5] Tuberous Sclerosis Alliance, http://tsalliance.org/pages.aspx?content=2
