In April 2020 the U.S. Food and Drug Administration (FDA) approved KOSELUGOTM (selumetinib) for use in children over age 2 with neurofibromatosis type 1 who have inoperable plexiform neurofibromas. This is the first drug ever approved specifically for a complication of NF Type 1. Obviously this has been huge news!
The process of getting this drug tested and approved has been a long road full of many collaborations including those between scientists and researchers as well as between NF organizations, volunteers and donors. The research was led by the National Cancer Institute (NCI), a division of the National Institutes of Health (NIH) with clinical trials conducted there in collaboration with clinical trials at several pediatric hospitals. Also, much of the funding came either through the federal government budget for the NIH or from the Congressionally Directed Medical Research Program (CDMRP). NF Midwest, NF Northeast, Texas NF and several other NF organizations and volunteers collaborated over the years to advocate for federal funds for NF research through these programs.
As NF regional patient organizations dedicated to informing and educating our communities, NF Midwest, NF Northeast and Texas NF collaborated along with several NF experts to bring you Frequently Asked Questions (FAQ) document on KOSELUGOTM .
UPDATE: As of November, 2025 Koselugo has been approved for adults.
Responses