In a landmark move for healthcare in the region, the United Arab Emirates has officially approved a groundbreaking gene therapy for a severe genetic disorder. The country's regulatory body has given the green light to Itvisma, known scientifically as onasemnogene abeparvovec, for treating spinal muscular atrophy in both adults and children aged two years and above.
A Pioneering Authorization in Global Healthcare
This decisive action by the Emirates Drug Establishment (EDE) positions the UAE as only the second country in the world to authorize this advanced treatment. The approval marks a significant leap forward, offering a new ray of hope for patients and families grappling with SMA while solidifying the UAE's reputation for fast-tracking access to cutting-edge medical solutions.
The therapy operates at the most fundamental level by addressing the root genetic cause of the disease. Itvisma works by replacing the defective or missing gene responsible for SMA, which is crucial for survival of motor neurons. This one-time treatment aims to halt disease progression, improve motor function, and significantly reduce the need for long-term, burdensome therapies.
Scientific Rigor and a Commitment to Patients
The EDE's approval was not granted lightly. It was based on a thorough review of compelling clinical evidence demonstrating clear and sustained improvements in patients' motor abilities. The data also confirmed a positive safety profile throughout all stages of the treatment's evaluation.
Dr. Fatima Al Kaabi, the Director General of the EDE, stated that this decision powerfully reflects the nation's dedication to providing patients with the latest genetic therapies, especially for rare diseases with high unmet needs. She highlighted that the UAE's regulatory model successfully balances speed, transparency, and safety, ensuring innovative treatments reach those in need both reliably and promptly.
A Milestone for Treatment and Regional Healthcare Leadership
For Novartis, the pharmaceutical giant behind Itvisma, the UAE's approval represents a major milestone in transforming patient care. Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, emphasized that close collaboration with the EDE and local partners will be key to enabling fast and equitable access for eligible patients. He noted that this support for advanced treatments can fundamentally alter the disease trajectory for individuals with SMA and aligns with Novartis's strategic focus on neurology and neuromuscular diseases.
Beyond immediate patient impact, this authorization significantly strengthens the UAE's position as a regional and global hub for pharmaceutical innovation. It underscores the nation's commitment to building a world-class, knowledge-driven healthcare system that prioritizes sustainability and, most importantly, improves the quality of life for patients across the Gulf and beyond.
