Italy approves reimbursement for mirdametinib to treat neurofibromatosis type 1

The Italian Medicines Agency (AIFA) has approved the reimbursement of mirdametinib (Ezmekly) for the treatment of symptomatic and inoperable plexiform neurofibromas associated with neurofibromatosis type 1 (NF1). The decision, applicable to pediatric and adult patients aged 2 years and older, makes the therapy available through the National Health Service.

Mirdametinib acts as a selective inhibitor of Mek 1/2 proteins to block tumor cell proliferation. Clinical data from the Phase 2b Reneu study showed an objective response rate of 41% in adults and 52% in pediatric patients, with significant reductions in tumor volume, pain, and functional deficits.

Merck officials noted that the approval provides a critical alternative to surgery, which is often difficult for deep or infiltrating lesions. Experts from the Gemelli University Hospital emphasized that the drug offers new opportunities for adults, who previously had limited therapeutic options beyond surgical intervention.