AIFA approves reimbursement for first topical gene therapy for butterfly children

The Italian Medicines Agency (AIFA) has approved the reimbursement of beremagene geperpavec (Vyjuvek) for the treatment of wounds in patients suffering from dystrophic epidermolysis bullosa. This rare genetic skin disorder, often referred to as “butterfly syndrome” due to the extreme fragility of the skin and mucous membranes, causes constant blistering and ulceration.

Recognized for its therapeutic innovation, the orphan drug is a topical gel applied once a week directly to lesions by healthcare professionals or trained caregivers. The treatment has demonstrated efficacy in healing wounds caused by the condition. Following this decision, the treatment will be covered by the Italian National Health Service (SSN).

In the same session, AIFA approved nine therapeutic options, including an anticancer drug, five indication extensions, and two equivalent medicines.