The UAE approved Itvisma gene therapy for SMA, improving motor function and reducing long-term treatments/Image: WAM
The UAE has taken a major step in healthcare innovation by approving Itvisma (onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy (SMA) in adults and children aged two years and above. This makes the country only the second in the world to authorize the treatment, offering new hope to patients and their families while reinforcing the UAE’s leadership in fast tracking access to advanced medical therapies.Itvisma works at the genetic level, replacing the defective gene responsible for SMA, which helps improve patients’ motor function and reduces dependence on long-term treatments. The Emirates Drug Establishment (EDE) granted the approval based on clinical evidence showing clear and sustained improvements in motor abilities and a positive safety profile throughout all stages of evaluation. Dr. Fatima Al Kaabi, Director General of EDE, said the decision reflects the UAE’s commitment to providing patients with cutting edge genetic therapies, particularly for rare diseases, while highlighting the efficiency and scientific rigor of the country’s regulatory system. She emphasized that the UAE’s model balances speed, transparency, and safety, ensuring that innovative treatments reach patients in a timely and reliable manner. For Novartis, the manufacturer of Itvisma, the approval is a milestone in patient care. Mohamed Ezz Eldin, Head of the GCC Cluster, noted that close collaboration with EDE and partners will enable fast and equitable access for patients who previously could not benefit from one-time gene therapy. He added that the UAE’s support for advanced treatments can change the disease trajectory of SMA and aligns with Novartis’s broader focus on neurology, neurodegenerative disorders, and neuromuscular diseases.The authorization of Itvisma also strengthens the UAE’s position as a regional hub for pharmaceutical innovation, delivering therapies that meet the highest standards of quality and safety. The decision underscores the nation’s dedication to building a state of the art healthcare system driven by knowledge, efficiency, and sustainability while improving the lives of patients across the region.
