AstraZenecahas announced thatKoselugo(selumetinib), an oral, selective MEK inhibitor, has been recommended for approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from KOMET, the largest and only placebo-controlled global Phase III trial in this patient population, which were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet. In the primary analysis of the trial, Koselugo showed a statistically significant objective response rate (ORR) of 20% (n=14/71, 95% CI: 11.2, 30.9) compared to 5% with placebo (n=4/74, 95% CI: 1.5, 13.3; p=0.01) by cycle 16.1 NF1 is a rare, progressive, genetic condition usually diagnosed in early childhood, but often progressing into adulthood, that can impact every organ system.2,3 Up to 50% of people living with NF1 may develop a type of non-malignant tumour called PN that may affect the brain, spinal cord and nerves. PN may appear later in a person’s life and can grow and become large, leading to pain, disfigurement and muscle weakness, among other debilitating symptoms. Prof. Pierre Wolkenstein, MD, PhD, Head of the Department of Dermatology at Henri Mondor Hospital, APHP, Paris East University (UPEC), and National Coordinating Investigator of the KOMET trial in Europe, said, “For adults with NF1, tumour growth doesn’t stop at childhood and can rapidly progress or develop into adulthood, impacting physical, emotional and social well-being. The positive recommendation by the CHMP for Koselugo in adults underscores the urgent need for additional targeted treatments for this patient population. When approved, Koselugo could offer a treatment option for adult patients and continuity of care across age groups, supported by established clinical experience and practice among doctors managing this lifelong condition.” Marc Dunoyer, Chief Executive Officer, Alexion, said, “The CHMP positive opinion of Koselugo in adults with NF1 PN builds on more than a decade of global clinical and real-world experience, reflecting the unmatched body of evidence supporting the safety profile and efficacy of Koselugo. As demonstrated by the results from KOMET, the largest and only placebo-controlled, double-blind global Phase III trial in an adult population, we continue to advance the pioneering legacy of Koselugo, which set the treatment standard in NF1 PN, to reach even more people worldwide.” The safety profile of Koselugo in the KOMET Phase III trial was consistent with its known profile and established use in paediatric patients. Koselugo has been recently approved in Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN based on data from the KOMET Phase III trial, and additional regulatory reviews are ongoing.