AIFA rare disease treatment approvals in Italy

The Italian Medicines Agency (AIFA) has expanded the reimbursement of several specialized therapies through the National Health Service.

On October 29, 2026, AIFA approved reimbursement for beremagene geperpavec (Vyjuvek), a topical gene therapy used to treat wounds in patients with dystrophic epidermolysis bullosa. This rare genetic condition, sometimes called “butterfly syndrome,” causes extreme skin fragility and constant blistering. The treatment is a gel applied weekly to lesions.

By November 2, 2026, AIFA announced additional reimbursements. These include Ezmekly (mirdametinib) for treating symptomatic and inoperable plexiform neurofibromas in patients with neurofibromatosis type 1, as well as tisotumab vedotin for adult patients with recurrent or metastatic cervical cancer. The agency also approved an indication extension for nintedanib to treat specific interstitial lung diseases in pediatric patients.

Regarding the Ezmekly approval, the agency specified that the therapy is authorized as a monotherapy for pediatric and adult patients with neurofibromatosis type 1 aged two years and older. This decision, published in the Official Gazette, aims to provide an alternative to surgery, which has previously been the primary option for adults with the condition and often carries risks of disfigurement or high recurrence rates. Experts note that mirdametinib offers a significant opportunity to reduce tumor volume and manage associated clinical symptoms.

Clinical data from the Phase 2b Reneu study indicated an objective response rate of 41% in adults and 52% in pediatric patients, showing significant reductions in pain and functional deficits. Merck officials and experts from Gemelli University Hospital noted that the drug provides a critical alternative for deep or infiltrating lesions where surgery is difficult.