(dailyRx News) A rare type of brain tumor called SEGA (subependymal giant cell astrocytoma) can appear in children. The drug designed to take on this cancer is too strong for young patients. Now a new children’s dosage of a medication has been approved.
The U.S. Food and Drug Administration has approved Afinitor Disperz (everolimus tablets for oral suspension). This is a new pediatric dosage form of Afinitor (everolimus). The medication is used to treat SEGA.
This is the first medicine to have an approved child dosage for treating a pediatric tumor.
Afinitor Disperz is recommended for patients with in-operable SEGA who are 1 year old and older with tuberous sclerosis complex (TSC).
Before this new dosing approval, Afinitor was recommended only for patients 3 years old and older. Afinitor received 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, MD, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research.
“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,” Dr. Pazdur said in a press release.
Afinitor Disperz dissolves easily in a small amount of water, making it easy to give to children who can't swallow whole tablets.
Afinitor’s manufacturer, Novartis, has been conducting ongoing studies to make sure the medication is safe and effective. This is a requirement of accelerated approval.
The company reported the results of a more recent study with 117 pediatric and adult patients. Participants received either Afinitor or a placebo daily.
Tumors shrunk in 35 percent of the patients who took Afinitor. None of the tumors got smaller among those who were treated with placebo.
According to the FDA, “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 of the medicine were mouth ulcers and respiratory tract infections.
Everolimus is the active ingredient in Afinitor and Afinitor Disperz. It blocks the uncontrolled activity of a protein called the mTOR kinase, which plays a critical role in the development and growth of SEGA tumors in patients with TSC.
The FDA has previously approved Afinitor to treat:
- adults with advanced renal cell carcinoma that has gotten worse after treatment with other 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).