Koselugo approved in Japan for paediatric patients with plexiform neurofibromas in neurofibromatosis type 1
First medicine approved in Japan to treat this rare and debilitating genetic condition. Pivotal SPRINT trial showed Koselugo reduced tumour volume.Koselugo (selumetinib) has been approved in Japan for the treatment of paediatric patients three years of age and older with plexiform neurofibromas (PNs) in neurofibromatosis type 1 (NF1) with clinical symptoms, such as pain and disfigurement, and PNs which cannot be completely removed by surgery without risk of substantial morbidity.[1] The approval by the Japanese Ministry of Health, Labour and Welfare (MHLW) is based on positive results