FDA Expands KOSELUGO Approval: Selumetinib for Adult NF1 Patients

Neurofibromatosis type 1 (NF1) is a genetic disorder affecting approximately 1 in 3,000 individuals, characterized by the development of benign tumors, including plexiform neurofibromas (PN)—complex, infiltrative growths that often cause significant morbidity such as pain, disfigurement, functional impairment, and reduced quality of life. In adults, symptomatic, inoperable PN represent a particularly challenging subset, as surgical resection is frequently infeasible due to tumor encasement of vital structures, invasiveness, or vascularity, leaving limited therapeutic options.