KOSELUGO demonstrates significant efficacy in reducing tumor burden in pediatric NF1 patients with inoperable plexiform neurofibromas.

Clinical Efficacy of KOSELUGO in NF1-Associated Plexiform Neurofibromas

KOSELUGO has shown a clinically meaningful therapeutic effect in pediatric patients with NF1 and symptomatic, inoperable plexiform neurofibromas. In pivotal clinical trials, the overall response rate was 66%, defined as at least a 20% reduction in tumor volume confirmed by imaging. The median time to response was 7.2 months, with a substantial proportion of patients maintaining response for 24 months or longer. KOSELUGO offers a targeted treatment option for a population with limited alternatives, improving morbidity associated with tumor burden. Efficacy in other populations or tumor types is not specified in the description.