AstraZeneca's Koselugo Approved in the EU for NF1 PN Treatment

AstraZeneca PLC announced that Koselugo (selumetinib) has been approved in the European Union for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1). The approval is based on results from the KOMET Phase III trial, which demonstrated a 20% objective response rate in tumour size reduction. The trial, the largest and only placebo-controlled global Phase III trial in this patient population, showed significant tumour volume reduction with a safety profile consistent with its established use in paediatric patients. This approval extends the life-changing potential of Koselugo to adults with NF1 PN in Europe, addressing a significant unmet need in the rare disease community.