Alexion, AstraZeneca Rare Disease, announced that the European Commission (EC) has approved Koselugo® (selumetinib), an oral, selective MEK inhibitor, for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1).
This approval follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) and is supported by results from KOMET, the largest and only placebo-controlled global Phase III trial conducted in this population. The results were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet, marking a key milestone in the management of this rare disease.
NF1 is a progressive genetic disorder that typically manifests in early childhood and can affect multiple organ systems throughout life. Up to 50% of people with NF1 develop plexiform neurofibromas, which are non-malignant tumors that can involve the brain, spinal cord, and peripheral nerves. These tumors often grow over time, causing pain, disfigurement, functional impairment, and other serious complications that severely affect quality of life.
“The approval of Koselugo for adults with NF1 PN in Europe offers patients and physicians a meaningful approach to close treatment gaps beyond childhood,” said Prof. Pierre Wolkenstein, MD, PhD, Head of Dermatology at Henri Mondor Hospital, Paris East University, and European Lead Investigator of the KOMET trial. “As demonstrated in the KOMET Phase III trial, the most robust late-stage clinical trial conducted in this patient group to-date, adults administered Koselugo saw significant tumour volume reduction with a safety profile consistent with its established use in paediatric patients, validating the clinical benefits of Koselugo for newly diagnosed adults and those transitioning to adult care.”
Marc Dunoyer, Chief Executive Officer of Alexion, added: “The European Commission approval extends the life-changing potential of Koselugo to adults with NF1 PN in the region, including continuity of care into adulthood. This milestone, along with our pioneering leadership in NF1 PN treatment landscape, embodies Alexion’s unwavering commitment to addressing the unmet needs in the rare disease community. We look forward to bringing Koselugo to those adults in need across Europe as soon as possible.”
In the primary analysis of the KOMET trial, Koselugo demonstrated a statistically significant objective response rate (ORR) of 20% (n=14/71; 95% CI: 11.2–30.9) compared with 5% in the placebo group (n=4/74; 95% CI: 1.5–13.3; p=0.01) at cycle 16. After 12 cycles, patients on placebo were switched to Koselugo, while those already receiving Koselugo continued treatment for another 12 cycles.
The safety profile of Koselugo was consistent with prior studies and its established use in paediatric patients. The therapy has also recently received regulatory approvals in Japan and other countries for the same indication, with additional reviews ongoing globally.
This latest EC approval represents a major step forward in expanding treatment access and improving outcomes for adults living with NF1-associated plexiform neurofibromas across Europe.
Also Read