**Summary**
AstraZenecas Koselugo (selumetinib) has been recommended for approval in the European Union (EU) by the Committee for Medicinal Products for Human Use (CHMP) for treating symptomatic, inoperable plexiform neurofibromas (PN) in adults with neurofibromatosis type 1 (NF1). This recommendation is based on positive results from the KOMET Phase III trial, which demonstrated a statistically significant 20% objective response rate (ORR) in tumor size reduction compared to 5% with placebo. KOMET is the largest and only placebo-controlled, double-blind global Phase III trial in this patient population, addressing a significant unmet need.
NF1 is a rare, progressive genetic condition that can lead to the development of PN, causing pain, disfigurement, and other debilitating symptoms. Koselugo, an oral MEK inhibitor, slows tumor growth by blocking overactive enzymes in NF1. The drug has already been approved in several countries, including Japan, for adult NF1 patients with symptomatic, inoperable PN, and is also approved for certain pediatric NF1 patients in the US, EU, Japan, China, and other regions.
The CHMPs positive opinion highlights the urgent need for targeted treatments in adult NF1 patients and underscores Koselugos established safety and efficacy profile. The drug’s approval could provide continuity of care across age groups, supported by over a decade of clinical and real-world experience. Additional regulatory reviews are ongoing in other regions.
AstraZeneca, through its rare disease division Alexion, continues to advance Koselugos pioneering legacy, aiming to reach more patients worldwide. The company’s strategic collaboration with MSD (Merck & Co.) further supports the development and commercialization of Koselugo and other innovative medicines.