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News From the Food and Drug Administration
May 19, 2020

A New Treatment for Children With Neurofibromatosis Type 1

JAMA. 2020;323(19):1887. doi:10.1001/jama.2020.7157

Selumetinib is the first drug approved for pediatric patients aged 2 years or older with neurofibromatosis type 1 (NF1), a rare disfiguring disease.

Marketed as Koselugo, the drug is a kinase inhibitor that helps stop neurofibroma tumor growth by blocking a key enzyme. It is indicated specifically for patients with symptomatic, inoperable plexiform neurofibromas, which involve the nerve sheaths and can grow anywhere in the body, including around the spine and in areas where they may affect internal organs. Caused by a genetic abnormality, NF1 is a progressive disease that’s usually diagnosed in childhood and affects about 1 in every 3000 infants.