A milestone for children with cancer and their families! The U.S. Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab), which is just the third drug in history to receive initial approval for treatment of pediatric cancer. It is the first immunotherapy drug for children with cancer.
Pediatric oncologist John Maris, MD, of the Cancer Center at The Children’s Hospital of Philadelphia, co-led the Phase 3 multicenter clinical trial of Unituxin to treat a form of high-risk neuroblastoma. After results of the trial were published in the New England Journal of Medicine, the standard treatment for this rare pediatric cancer was changed to include use of this new drug.
Unituxin has been proven to increase survival by as much as 15 percent in children with high-risk neuroblastoma, a cancer which attacks the peripheral nervous system.
It is unusual for a drug to receive FDA approval so quickly. But as a result of the Creating Hope Act of 2012, which encourages drug development in treating children’s diseases, the FDA created a program to speed up the approval process for qualified therapies. For this reason, the manufacturer of Unituxin, United Therapeutics, received a pediatric priority review voucher from the FDA.
Unituxin is particularly exciting because it’s the first pediatric cancer therapy to prove that immunotherapy works.
Immunotherapeutics, which are treatments that use the body’s own immune system to fight disease, have long shown promise in clinical trials. A $14.5 million grant, from Stand Up 2 Cancer — St. Baldrick’s Foundation to the Pediatric Dream Team, has allowed physician scientists to translate years of immunotherapy research from the laboratory to bedside.
The hope is to one day have immunotherapy drugs designed to treat other types of pediatric cancers, which will treat these devastating diseases more effectively and with fewer side effects.