The UAE’s Emirates Drug Establishment (EDE) has approved Itvisma (onasemnogene abeparvovec), a gene therapy that uses an adeno-associated viral vector to treat spinal muscular atrophy (SMA) in eligible adults and children aged two years and above.
With this decision, the UAE becomes the second country globally to grant approval for Itvisma gene therapy, reinforcing its leadership in accelerating patient access to advanced medical innovations across the region.
Itvisma targets the genetic cause of spinal muscular atrophy by replacing the defective gene responsible for the disease, helping to improve patients’ motor functions and reduce their reliance on long-term treatments.
This regulatory authorization is part of EDE’s ongoing commitment to enabling access to high-value, advanced therapies and accelerating the delivery of breakthrough medicines to UAE patients, in line with the UAE’s vision for a world-class, efficient, innovative, and sustainable healthcare ecosystem.
Dr. Fatima Al Kaabi Director-General Emirates Drug Establishment
“Granting the regulatory approval for Itvisma is a significant step demonstrating the UAE’s commitment to providing patients with the latest advanced genetic therapies, particularly those with rare genetic diseases like spinal muscular atrophy. This decision showcases the efficiency of the country’s health system and its capability to evaluate and approve innovative drugs according to the highest scientific standards within effective timeframes. We are committed to ensuring patients receive rapid access to cutting-edge treatment options that improve their quality of life.”
Al Kaabi clarified that Itvisma was approved following clinical evidence that resulted in clear and sustained improvements in patients’ motor abilities, in addition to a consistently positive safety profile observed throughout all stages of clinical evaluation. She added that the Emirates Drug Establishment remains committed to supporting medical innovation and adopting modern therapies that enhance quality of life, aligned with the UAE’s vision for a world-class, sustainable healthcare sector.
Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, stressed that the authorization of Itvisma in the UAE is first and foremost about serving patients and their families. He pointed out that close cooperation with the Emirates Drug Establishment and partners aims to ensure fast, equitable access to the treatment, providing people living with SMA who were previously unable to benefit from a one-time gene therapy a new opportunity to receive this type of medical intervention.
Rep. Image credits: freepik | Cropped by GBN
The GCC Head noted that this achievement reflects Novartis’s commitment to neurology, building on its extensive experience in treating SMA and multiple sclerosis, while expanding its focus into neuroimmunology, neurodegenerative disorders, and neuromuscular diseases.
Itvisma’s approval is a strategic milestone within the Emirates Drug Establishment’s mission to position the UAE as a regional hub for pharmaceutical innovation by delivering advanced genetic therapies that meet the highest standards of quality and safety.
