The Emirates Drug Establishment has approved Itvisma, a groundbreaking gene therapy for the treatment of spinal muscular atrophy, marking a major milestone in the UAE’s efforts to accelerate access to advanced medical innovations for patients with rare diseases.
With the decision, the UAE becomes only the second country worldwide to authorise the therapy, reinforcing its position as a leader in fast-tracking high-value treatments through a robust and science-led regulatory framework.
Itvisma, also known as onasemnogene abeparvovec, targets the root genetic cause of spinal muscular atrophy by replacing the faulty gene responsible for the condition. The therapy is approved for eligible adults and children aged two years and above, offering the potential for improved motor function and reduced dependence on long-term treatment.
The approval aligns with the Emirates Drug Establishment’s strategy to support rapid access to breakthrough therapies while maintaining the highest standards of safety, quality, and scientific rigour. Clinical evidence reviewed during the evaluation process demonstrated sustained improvements in patients’ motor abilities alongside a consistently positive safety profile.
Dr. Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, said the authorisation reflects the UAE’s commitment to delivering cutting-edge genetic therapies and strengthening international confidence in its regulatory model.
Novartis, the developer of Itvisma, highlighted that close collaboration with UAE health authorities will help ensure equitable and timely access for patients and families affected by spinal muscular atrophy.
The approval is seen as a strategic step in positioning the UAE as a regional hub for pharmaceutical innovation and advanced genetic medicine.
News Source: Emirates News Agency
