L'OMS: les enfants gravement malades reçoivent un médicament important

The Croatian Health Insurance Fund (HZZO) has announced that its Drug Committee has issued a positive opinion regarding the inclusion of a medication containing the active substance givinostat on the list of reimbursable drugs for treating Duchenne muscular dystrophy. The decision comes after extensive evaluation and reflects a significant step toward making this treatment accessible to affected children in Croatia. The medication, which slows disease progression, would be fully funded through mandatory health insurance if approved. It would be available under the same conditions as other EU member states, following approval from the European Commission and the European Medicines Agency. Givinostat is intended for the treatment of ambulatory boys with Duchenne muscular dystrophy aged six and older who are undergoing corticosteroid therapy. This marks a crucial development for families dealing with the condition, offering hope for delayed loss of mobility and reduced dependence on wheelchairs. According to HZZO officials, the decision was made in response to public interest in the topic. They emphasized that the committee’s positive opinion will be forwarded to the Board of Directors of the HZZO, which will make the final decision on including the drug on the reimbursement list. The process follows established regulations and ensures transparency and adherence to legal frameworks governing pharmaceutical approvals. Duchenne muscular dystrophy is a severe genetic disorder characterized by progressive muscle degeneration. In Croatia, approximately 60 boys are diagnosed with the condition. Of these, around 20 meet the criteria for givinostat treatment, while four are in particularly severe health conditions. Without timely intervention, these children face permanent loss of independent walking ability and eventual reliance on wheelchairs. The potential availability of givinostat could significantly alter their long-term prognosis. The decision to release the committee's opinion before the final board vote underscores the urgency of the situation and the importance of providing access to innovative treatments. HZZO officials have stressed that the ultimate approval rests with the board, which will consider all relevant factors before making its determination. The timing of the decision aligns with broader efforts to improve access to cutting-edge therapies for rare diseases within the country. Health professionals and patient advocacy groups have welcomed the move, highlighting the critical need for effective interventions in cases of severe neuromuscular disorders. While the final approval is still pending, the positive opinion represents a major milestone. Families of affected children are hopeful that the treatment will soon become available, allowing them to pursue better outcomes for their loved ones. The outcome of the board’s deliberation will determine whether givinostat joins the list of medications covered by Croatia’s national healthcare system.