In a landmark medical advancement, the United Arab Emirates has approved Itvisma, a cutting-edge gene therapy developed by Swiss pharmaceutical company Novartis, to treat spinal muscular atrophy (SMA) in adults and children aged two years and older. The approval positions the UAE as only the second country in the world to authorise this advanced treatment after the United States. This decision highlights the nation’s commitment to embracing breakthrough therapies and expanding access to life-changing medical innovations for patients living with rare genetic diseases.
Itvisma works by replacing the defective SMN1 gene responsible for SMA, offering the potential to improve motor function and reduce long-term reliance on chronic treatments. Delivered in a single fixed dose, this therapy marks a significant shift in how SMA is managed, especially for older children, teens and adults who previously had limited treatment options.
This article explores what SMA is, how Itvisma works, the significance of this approval in the UAE, where it fits within global medical innovation, and what it means for patients and families facing this challenging disease.
Understanding Spinal Muscular Atrophy
Spinal muscular atrophy is a rare genetic neuromuscular disorder caused by a mutation or defect in the survival motor neuron 1 (SMN1) gene, which is responsible for producing a protein essential for the survival of motor neurons. Without sufficient SMN protein, motor neurons begin to degenerate, leading to progressive muscle weakness and atrophy that can affect breathing, swallowing and movement.
SMA varies in severity, with Types 1 to 4 describing a spectrum from the most severe infant form to milder adult onset forms. The disease progressively weakens motor skills and, in its most severe forms, can be life threatening within the first years of life if left untreated.
Until recent years, treatment options focused on supportive care and therapies that helped increase levels of SMN protein but did not address the underlying genetic cause of the disease. The approval of gene therapies such as Itvisma marks a new era in directly targeting the root cause of SMA.
How Itvisma Gene Therapy Works
Itvisma (onasemnogene abeparvovec) is a gene replacement therapy designed to deliver a working copy of the SMN1 gene directly to the patient’s cells. The therapy uses a harmless viral vector to introduce the functional gene, enabling cells to begin producing the SMN protein needed for proper motor neuron function.
Unlike other treatments that may require repeated dosing over time, Itvisma is given in a single fixed dose, regardless of body weight or age. This one-time administration sets it apart from many traditional therapies and offers a possible long-lasting benefit, reducing the need for chronic treatment regimens.
Clinical trials and regulatory reviews have shown that Itvisma can lead to improved motor function and stabilization of SMA progression in patients. These findings underpinned its earlier approval by the US Food and Drug Administration (FDA) and now its regulatory acceptance in the UAE.
Significance of UAE Approval
The Emirates Drug Establishment (EDE) granted regulatory approval for Itvisma in the UAE following a thorough evaluation of clinical evidence demonstrating sustained improvements in motor abilities and a positive safety profile.
Director-General Dr Fatima Al Kaabi emphasised that the approval reflects the UAE’s commitment to providing patients with the latest and most effective genetic therapies. Authorities highlighted the country’s ability to evaluate and authorise innovative treatments in line with high scientific standards, ensuring that patients gain rapid access to cutting-edge options that can improve quality of life.
The UAE’s decision makes it the second nation globally to approve Itvisma for medical use in both adults and pediatric patients aged two and above, following its earlier FDA approval in the United States. This rapid adoption underscores the nation’s position as a leader in accelerating access to advanced medical innovations.
Impact on Patients and Families
For patients living with SMA and their families, the approval of Itvisma represents renewed hope. The therapy offers the possibility of improved motor function, reduced disease progression and less reliance on long-term treatments that have previously dominated SMA care.
Parents and caregivers have faced years of uncertainty and intensive care routines for affected children. With Itvisma, a single dosing model could significantly reduce the treatment burden and lead to sustained improvements in mobility and independence for many patients.
While cost and availability details in the UAE have not yet been finalised, the approval sets the stage for healthcare providers to begin integrating this transformative therapy into treatment plans for eligible SMA patients.
The UAE’s Healthcare Innovation Landscape
The UAE has been steadily expanding its role as a regional hub for pharmaceutical innovation and advanced medical therapies. Approving Itvisma aligns with a broader strategy to adopt breakthroughs in genetic medicine, rare disease treatment and precision healthcare technologies.
Health authorities have emphasised the importance of balancing scientific rigor with timely regulatory action to ensure that patients benefit from global advancements in treatment. The UAE’s regulatory framework supports rapid evaluation while upholding high safety and efficacy standards.
This regulatory approach not only benefits local patients but also strengthens the country’s reputation as a leader in healthcare innovation, attracting global partnerships and fostering a vibrant ecosystem for advanced medical research and clinical care.
How Itvisma Fits Into Global SMA Treatment Trends
The approval of Itvisma in the UAE follows a global trend toward embracing gene therapies that target the underlying causes of genetic diseases rather than simply managing symptoms. After its initial FDA approval in late 2025, Itvisma represented a significant evolution beyond earlier SMA treatments such as Spinraza and Evrysdi, which modulate gene expression but do not replace the defective SMN1 gene.
Gene replacement therapies like Itvisma and its predecessor Zolgensma have shifted the paradigm of SMA care by offering one-time, targeted treatments that can change the disease course. These therapies have shown meaningful improvements in motor function and survival outcomes, particularly when administered early in the disease process.
The UAE’s approval emphasizes its alignment with global best practices in rare disease treatment and reflects confidence in the scientific evidence supporting these transformative therapies.
Challenges and Considerations
Despite the excitement surrounding Itvisma’s approval, several challenges remain. Gene therapies like Itvisma are often extremely costly, with prices reported in the millions of dollars for a single dose. This raises questions about accessibility and reimbursement, particularly for patients without comprehensive insurance coverage or public funding support.
Healthcare providers, insurance systems and government programmes will need to work collaboratively to ensure equitable access to this treatment. Decisions on pricing structures, reimbursement policies and patient assistance programmes will influence how broadly the therapy can reach those in need.
Long-term monitoring of treatment outcomes and safety will also be critical as more patients receive Itvisma, helping clinicians learn more about real-world effects and optimize care strategies.
Patient Stories Highlight the Need for Innovation
Human stories illustrate the profound impact of genetic therapies. For example, in mid-2025 the Ruler of Dubai, Sheikh Mohammed bin Rashid, funded a Dh7 million Itvisma treatment for a young Syrian girl with SMA, demonstrating the potential of gene therapy to change lives and the importance of public support in ensuring access to advanced care.
Such cases underscore not only the medical potential of therapies like Itvisma but also the emotional and societal importance of making breakthrough treatments available to vulnerable populations.
Looking Ahead: Future of SMA Care in the UAE
With regulatory approval secured, the next steps involve making Itvisma available clinically, ensuring healthcare professionals are trained in its administration, and establishing appropriate support systems for long-term patient follow-up.
Medical centres in the UAE that specialise in neurology, paediatrics and gene therapy will likely play leading roles in delivering and evaluating outcomes for SMA patients receiving Itvisma. Collaborative research, data sharing and integration with global SMA care networks will support continuous learning and improve care standards over time.
As more advanced therapies come into regulatory pipelines, the UAE’s decision to embrace gene therapy today puts it at the forefront of delivering next-generation care for rare genetic diseases.
Conclusion
The UAE’s approval of the Itvisma gene therapy for spinal muscular atrophy marks a major achievement in healthcare innovation and rare disease treatment. By offering a single-dose, targeted therapy to both children and adults with SMA, the country is expanding access to transformative medical care that addresses the root genetic cause of this debilitating disease.
As global recognition of gene therapies continues to grow, the UAE’s proactive regulatory stance highlights its leadership in adopting cutting-edge medical advancements. Patients and families affected by SMA now have new cause for hope, and the broader healthcare system has taken a meaningful step toward integrating the most advanced therapies in modern medicine.
