The UAE has approved the use of gene replacement therapy for a new age group of patients living with spinal muscular atrophy (SMA), strengthening the country’s position as a regional leader in access to advanced medical innovation.
The Emirates Drug Establishment (EDE) announced the regulatory approval of Itvisma (onasemnogene abeparvovec), a one-time gene therapy for eligible adults and children aged two years and above. With the decision, the UAE becomes the second country globally to authorise the treatment for this expanded patient group.
Itvisma addresses the root genetic cause of SMA by replacing the defective gene responsible for the condition. The therapy has been shown to improve motor function and reduce reliance on long-term supportive treatments.
UAE approves expanded SMA gene treatment
The approval forms part of EDE’s broader strategy to accelerate patient access to high-value and breakthrough medicines, in line with the UAE’s ambition to build a world-class, innovative and sustainable healthcare system.
Dr. Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, said the decision reflects the UAE’s commitment to patients with rare genetic diseases. “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,” she said.
She added that the approval highlights the efficiency of the national health system in evaluating innovative treatments 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 said.
According to EDE, the therapy was authorised based on clinical evidence showing clear and sustained improvements in patients’ motor abilities, alongside a consistently positive safety profile throughout clinical evaluation.
Al Kaabi noted that the decision reinforces international confidence in the UAE’s regulatory model, which balances speed, scientific rigour and transparency to ensure timely and reliable access to innovation. She added that EDE will continue supporting medical innovation that enhances quality of life and advances the country’s healthcare ecosystem.
Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, said the authorisation represents a meaningful step for patients and families affected by SMA. He emphasised that collaboration with EDE aims to ensure fast and equitable access to the therapy for those who were previously unable to benefit from a one-time gene treatment.
Ezz Eldin added that the UAE’s support for innovative medicines can help alter the disease trajectory for SMA patients, noting that the approval reflects Novartis’s continued commitment to neurological and neuromuscular disorders.
EDE said the milestone supports its mission to position the UAE as a regional hub for pharmaceutical innovation, delivering advanced genetic therapies that meet the highest standards of safety and quality.
