Selumetinib (granule formulation) for treating neurofibromas type 1-related symptomatic, inoperable plexiform in children

Selumetinib granule formulation is currently in clinical development for the treatment of symptomatic and inoperable plexiform neurofibromas (PN) associated with type 1 neurofibromatosis (NF1) in children. NF1 is a genetic condition that causes tumours to grow along the nerves. The tumours are usually non-cancerous but may cause a range of symptoms. Some children have a special kind of neurofibroma called PN. These often develop early in childhood involve swelling to the whole section of a nerve and its branches. There is currently no cure for NF1. Surgery carries several risks and a proportion of PN are considered inoperable. Children with NF1-associated PNs experience substantial disease burden with significant unmet needs and increased risk of developing other tumours. These tumours are typically located in challenging areas such as the head, neck, chest, and spine. In most cases, the skin is affected, causing symptoms such as birthmarks but the most severe symptoms include pain, disfigurement, and difficulties with movement.