The Children's Tumor Foundation
09/29/2025 | Press release | Archived content
FDA Expands Koselugo Approval to Younger Children with NF1
The U.S. Food and Drug Administration (FDA) has expanded the approval of Koselugo (selumetinib) to include children as young as 1 year old who are living with neurofibromatosis type 1 (NF1) and have symptomatic, inoperable plexiform neurofibromas (PN). Previously, Koselugo was only approved for children ages 2 and older.
This update means families and physicians now have the option to start treatment earlier, during a time when tumors can already cause significant challenges for children with NF1.
A first-of-its-kind treatment for NF1
When the FDA first approved Koselugo in April 2020, it was a landmark moment: the first-ever treatment approved for NF. Affecting 1 in 2,000 people, NF has long had no approved therapies. Koselugo's approval marked a turning point, giving patients and families the first tangible option for medical treatment of NF1 plexiform neurofibromas.
The original approval was based on clinical trials at the National Cancer Institute (NCI), part of the NIH, where more than 70% of children with NF1 and inoperable plexiform neurofibromas saw tumors shrink by 20-60%. Families also reported less pain, better mobility, improved function, and greater emotional well-being. The Children's Tumor Foundation (CTF) helped make these trials possible: CTF-funded researchers were the first to show that MEK inhibitors could shrink NF tumors, with promising results shared at the CTF NF Conference in 2015, and later published in the New England Journal of Medicine. These milestones laid the foundation for the NCI trials and the FDA's historic approval of Koselugo.
Moving forward
Koselugo's initial 2020 approval represented a breakthrough for NF1. This latest expansion builds on that milestone, reaffirming the importance of research and clinical trials in creating new treatment options. It also signals hope for the continued development of therapies for all forms of NF.
The Children's Tumor Foundation published this content on September 29, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on October 01, 2025 at 14:27 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]