Selumetinib Approved by FDA to Treat Children with NF1

April 14, 2020, by NCI Staff

Kristina hugs her daughter, Jane, who was treated with selumetinib as part of a clinical trial led by researchers in NCI's Pediatric Oncology Branch.
Credit: National Cancer Institute

The Food and Drug Administration (FDA) has approved selumetinib (Koselugo) to treat children with neurofibromatosis type 1 (NF1) and tumors called plexiform neurofibromas.

NF1 is a genetic disorder that causes patients to develop many types of tumors, including plexiform neurofibromas, which can form along the nervous system anywhere in the body. While plexiform neurofibromas are not cancerous, they frequently grow throughout childhood and can cause many clinical problems, including disfigurement, pain, and functional impairments.

Although plexiform neurofibromas can be removed surgically in some children, surgery isn’t an option for most because of the sensitive locations in which the tumors form. As a result, the approval is expected to have an immediate effect on patient care.

The approval was based on results from a clinical trial, designed and led by researchers in NCI’s Pediatric Oncology Branch (POB), which showed that the drug shrank neurofibromas in 70% of patients. For many children in the trial, treatment with selumetinib also had clinical benefit, improving their pain, function, and quality of life.

The approval was hailed by many families of children with NF1, including a tremendous outpouring on social media.

“Over 10 years of clinical trials, 4 rounds of experimental medicine, hundreds of blood draws, tens of thousands of miles traveled, weeks away from the family, helped to create this opportunity to make life better for children facing NF,” wrote Kelly Carpenter on Twitter. Her son, Travis, was treated by POB researchers at the NIH Clinical Center as part of the clinical trial.

Results from the trial were published March 18 in the New England Journal of Medicine. More information about the trial is available in this NCI news release on the trial findings and this June 2018 Cancer Currents story.