The United Arab Emirates has approved Itvisma (onasemnogene abeparvovec), a pioneering gene therapy by Novartis, for children and adults aged two and above with spinal muscular atrophy (SMA).
The therapy replaces the faulty SMN1 gene, tackling the root cause of the disease rather than just symptoms. Administered as a single dose, Itvisma has shown improvements in muscle strength and motor function.
Dr. Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, said the approval reflects the UAE’s commitment to rapid, safe access to advanced treatments. Novartis highlighted the therapy’s potential to transform the lives of SMA patients.