From GlobeNewswire: 2025-05-24 19:17:00

SpringWorks Therapeutics announced that the CHMP recommended conditional marketing authorization for mirdametinib in the treatment of symptomatic and inoperable NF1-NP. The final decision by the European Commission is expected in Q3 2025. NF1 affects 3 in 10,000 individuals in the EU, with about 135,000 people affected. The CHMP based its decision on the ReNeu phase 2b trial, showing a 41% objective response rate in adults and 52% in children, with significant improvements in pain and quality of life. Mirdametinib is a MEK inhibitor approved in the US for NF1 treatment.

NF1-NP is a genetic disorder with a high risk of developing plexiform neurofibromas, which can be aggressive and life-threatening. Surgical removal is challenging due to infiltrative growth along nerves. Mirdametinib, an oral MEK inhibitor, has demonstrated safety and efficacy in treating NF1. The ongoing ReNeu trial evaluated mirdametinib in patients with inoperable NF1-NP, showing significant reduction in tumor volume and improvements in patient-reported outcomes. SpringWorks Therapeutics aims to provide innovative therapies for rare diseases and cancer.



Read more at GlobeNewswire: SpringWorks Therapeutics recibe un dictamen positivo del