Posted by Michael Wonder on 11 Feb 2025
FDA approves mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection
11 February 2025 - Today, the FDA approved mirdametinib (Gomekli, SpringWorks Therapeutics), a kinase inhibitor, for adult and paediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.
Efficacy was evaluated in ReNeu, a multi-centre, single-arm trial in 114 patients ≥2 years of age (58 adults, 56 paediatric patients) with symptomatic, inoperable NF1-associated plexiform neurofibromas causing significant morbidity.
