Positive CHMP Opinion for Koselugo: A Breakthrough Treatment for Adults with NF1-Related Tumors

European Medicines Agency Moves Toward Approval of Koselugo for Adults with Neurofibromatosis Type 1

Brussels, Belgium — In a significant step forward for patients suffering from Neurofibromatosis Type 1 (NF1), the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for the use of Koselugo (selumetinib) in adults with symptomatic, inoperable plexiform neurofibromas (PN). This recommendation is based on groundbreaking results from the KOMET trial, the largest placebo-controlled global Phase III study conducted in this patient population.

The findings were unveiled at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and subsequently published in The Lancet. The primary analysis revealed a statistically significant objective response rate (ORR) of 20% for Koselugo, compared to just 5% for the placebo group, marking a pivotal moment in the treatment landscape for NF1.

NF1 is a rare genetic disorder that typically manifests in early childhood but can persist and worsen into adulthood, affecting multiple organ systems. Up to 50% of individuals with NF1 may develop non-malignant tumors known as plexiform neurofibromas, which can lead to severe complications, including pain, disfigurement, and muscle weakness.

Prof. Pierre Wolkenstein, MD, PhD, Head of the Department of Dermatology at Henri Mondor Hospital in Paris and the National Coordinating Investigator for the KOMET trial in Europe, emphasized the importance of this development. “For adults with NF1, tumor growth doesn’t stop at childhood and can rapidly progress, impacting physical, emotional, and social well-being. The CHMP’s positive recommendation for Koselugo underscores the urgent need for targeted treatments for this patient population,” he stated.

Marc Dunoyer, Chief Executive Officer of Alexion, the company behind Koselugo, expressed optimism about the future. “The CHMP’s positive opinion builds on over a decade of clinical and real-world experience, reflecting the robust evidence supporting Koselugo’s safety and efficacy. As demonstrated by the KOMET trial results, we are committed to advancing the pioneering legacy of Koselugo, which has set the treatment standard in NF1 PN, to reach even more patients worldwide.”

The safety profile of Koselugo observed in the KOMET trial aligns with its established use in pediatric patients, offering reassurance to healthcare providers and patients alike. Koselugo has already received approval in Japan and other countries for treating adult patients with NF1 who have symptomatic, inoperable PN, with additional regulatory reviews currently underway.

As the EMA moves closer to a final decision, the hope is that Koselugo will soon provide a much-needed treatment option for adults grappling with the challenges of NF1, ensuring continuity of care across age groups and improving the quality of life for many.