Plain language summary of the ReNeu study: a study of mirdametinib for adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas

Plain Language SummaryWhat is this summary about?This is a summary of the ReNeu study. The researchers in this study wanted to know if the drug mirdametinib, also known as GOMEKLI® and EZMEKLY®, could help adults and children 2 years and older with Neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (‘Plexiforms’), also known as NF1-PN.The study was designed to evaluate whether mirdametinib works and is safe in adults and children living with this disease. To see whether mirdametinib worked, the researchers assessed if it could: Reduce the size of plexiforms.Reduce pain.Improve health-related quality of life.This study included 58 adults and 56 children who had NF1 and a plexiform that was causing significant problems, such as pain, problems with mobility, difficulty breathing, or disfigurement. The participants’ plexiforms also could not be removed with surgery.What were the ReNeu study results?In the ReNeu study, mirdametinib helped some adults and children to have: Decreases in the size of their target plexiform.Reduced pain intensity, including a reduced impact of their pain on daily activities.Improved health-related quality of life.During this study, 98% of the adults (57 out of 58) and 95% of the children (53 out of 56) had side effects that the doctors considered to be related to mirdametinib. Most of these side effects were not serious.What do these study results mean?Mirdametinib helped to shrink plexiforms, reduce the intensity of the participants’ pain and how much the pain interfered with their daily life, and improve health-related quality of life in adults and children.The approval of mirdametinib by the FDA in the United States and by the EC in the European Union is based on the ReNeu study. These agencies have approved the use of mirdametinib to treat adults and children 2 years of age and older with NF1 who have plexiforms that cause symptoms and cannot be removed or can only be partially removed by surgery.Clinical trial number: NCT03962543