By: IPP Bureau
Last updated : September 24, 2025 9:42 am
The positive opinion is based on results from KOMET, the largest and only placebo-controlled, double-blind global Phase III trial in this patient population
AstraZeneca and Alexion announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Koselugo (selumetinib) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1).
The positive opinion is based on results from KOMET, the largest and only placebo-controlled, double-blind global Phase III trial in this patient population.
NF1 is a rare, progressive genetic condition, typically diagnosed in childhood but often persisting and progressing into adulthood. Up to 50 per cent of individuals with NF1 develop plexiform neurofibromas, non-malignant tumours that can affect the brain, spinal cord and peripheral nerves. These tumours may cause pain, disfigurement, muscle weakness and other debilitating symptoms, significantly impacting quality of life.
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 does not stop at childhood but can continue or develop later in life, severely affecting physical, emotional and social well-being. The CHMP’s positive recommendation for Koselugo underscores the urgent need for additional targeted treatments for this population. When approved, Koselugo could offer an important treatment option for adults while ensuring continuity of care across age groups.”
Marc Dunoyer, Chief Executive Officer, Alexion, added: “This recommendation builds on more than a decade of clinical and real-world experience with Koselugo, reinforcing its strong safety and efficacy profile. The KOMET Phase III trial further demonstrates Koselugo’s role in setting the treatment standard for NF1 plexiform neurofibromas. We are proud to advance this pioneering medicine to help more people living with this lifelong condition.”
Koselugo has already been approved in Japan and other countries for the treatment of adult patients with symptomatic, inoperable PN, based on the KOMET Phase III trial. Additional regulatory reviews remain ongoing globally.