AstraZeneca’s Koselugo (selumetinib) has secured a positive recommendation from the European Medicines Agency’s CHMP (Committee for Medicinal Products for Human Use) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1).
Clinical Evidence: The KOMET Phase III Trial
The CHMP’s endorsement is grounded in data from the KOMET Phase III trial, a global, placebo-controlled, double-blind study in adult NF1 patients. The trial demonstrated a ~20 % objective response rate (ORR) in tumour volume reduction over placebo, supporting the efficacy profile of Koselugo in adults.
Previous studies have already established Koselugo’s therapeutic value in paediatric NF1 PN, and this new recommendation expands its potential to adult populations.
Regulatory Landscape and Next Steps
If the European Commission grants approval based on the CHMP opinion, Koselugo would become available across EU member states for adult NF1 patients with symptomatic, inoperable PN.
AstraZeneca is in active regulatory dialogue across global jurisdictions to extend its label and ensure patient access in adult populations.
About Neurofibromatosis Type 1 & Plexiform Neurofibromas
NF1 is a genetic disorder characterised by nerve sheath tumours. Plexiform neurofibromas are benign but can cause pain, disfigurement, and functional impairment, and often cannot be removed surgically. There is significant unmet need, particularly in adult patients.
