2025 Year in Review: Neurofibromatosis Type 1 With Plexiform Neurofibromas

For patients with the rare condition neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN), 2025 was a breakthrough year. In February, FDA approved the kinase inhibitor mirdametinib (Gomekli) for the treatment of adult and pediatric patients aged 2 years and older based on the ReNeu trial, which found lasting reductions in PN volume, along with improvements in pain severity and quality of life. Mirdametinib also received conditional European approval. Experts urged pairing therapy with surgery and said research into the condition continues. In September, a second therapy, selumetinib (Koselugo), was approved in the pediatric population; in November, it was approved for adults.