FDA Approves First Therapy for Children with NF1

On April 10, 2020, the US Food and Drug Administration (FDA) approved the drug selumentinib (Koselugo) for people with Neurofibromatosis Type 1 (NF1) who are 2 years of age and older. The first of its kind drug will be used to treat symptomatic, inoperable plexiform neurofibromas. These tumors are often detected in young children, and may involve the eye socket, face, arm, leg, back, chest, or abdomen.
The FDA approval came after a clinical trial conducted by the National Cancer Institute showed that nearly two thirds of patients had a positive response to the drug, with at least a 20% reduction in tumor volume. Of these patients, most had responses that lasted 12 months or longer. However, no patients experienced complete disappearance of their tumor.

“These are exciting times for families affected with NF1”, says NF Center Director, David H. Gutmann, MD, PhD. “This success is the result of decades of hard work by scientists, physicians, nurses, and patient advocacy groups.”