The Children's Tumor Foundation
11/12/2025 | Press release | Distributed by Public on 11/12/2025 10:32
Koselugo Approved in the EU for Adults with NF1 Plexiform Neurofibromas
Koselugo Approved in the EU for Adults with NF1 Plexiform Neurofibromas
November 12, 2025Featured EU, Pharma, Science & Research
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The European Commission (EU) has approved Koselugo (selumetinib) for adult patients in the EU with symptomatic, inoperable plexiform neurofibromas (PN) associated with neurofibromatosis type 1 (NF1). The decision follows a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), which reviews and recommends medicines for approval in the EU. It is also supported by results from the KOMET Phase III trial, which showed a 20% tumor response rate compared to 5% with placebo.
Read the official press release from Alexion.
NF1 is a rare genetic condition that often begins in childhood and can lead to tumors on nerves throughout the body. These plexiform neurofibromas can cause pain and other serious complications. This EU decision expands treatment access to adults, following earlier authorization for children and adolescents with NF1-related PN.
CTF's Role in Koselugo's Development
The Children's Tumor Foundation (CTF) played a central role in paving the way for Koselugo, the first-ever approved treatment for NF1-related plexiform neurofibromas. CTF helped lay early groundwork by funding research that first identified MEK inhibitors as a promising therapeutic strategy for NF tumors. The Foundation worked with the National Cancer Institute (NCI), Neurofibromatosis Therapeutic Acceleration Program (NTAP), and other partners to move these findings into clinical trials. Learn more about CTF's role.
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