Koselugo Approved for Children with Neurofibromatosis Type 1

April 14, 2020

The FDA approved AstraZeneca’s and Merck’s Koselugo (selumetinib) for treatment of  neurofibromatosis type 1 (NF1) in pediatric patients two years and older, making it the first drug approved for the rare disease.

The drug is approved for patients who have symptomatic, inoperable neurofibromas. The tumors can grow anywhere in the body, including the face, extremities, around the spine and deep in the body where they may affect organs.

The agency reviewed the drug via multiple accelerated pathways, including priority review, breakthrough therapy, orphan drug and rare pediatric disease designations. 

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