The EU approval makes Koselugo (selumetinib) the first treatment option for children aged three and over with neurofibromatosis type 1 (NF1), a debilitating disease that affects one in 3,000 individuals worldwide.
Up to half of patients with NF1 develop the benign but problematic tumours – called plexiform neurofibromas – of the peripheral nerves. They can occur anywhere in the body, causing pain, motor dysfunction and lumps under the skin that in some cases can turn malignant.