UAE Becomes Second Country to Approve Gene Therapy Itvisma for Spinal Muscular Atrophy

The United Arab Emirates has become the second country in the world to approve the gene therapy Itvisma for the treatment of spinal muscular atrophy (SMA), marking another milestone in the global adoption of advanced genetic medicines. The approval applies to patients aged two years and older, according to reports released this week.

SMA is a rare, inherited neuromuscular disorder caused by the loss or mutation of the SMN1 gene, leading to progressive muscle weakness and, in severe cases, early mortality. Gene therapies aim to address the disease at its root by delivering a functional copy of the defective gene, offering the potential for long-term or permanent clinical benefit following a single administration.

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Itvisma works by using a viral vector to introduce a functional SMN gene into motor neurons, thereby restoring the production of survival motor neuron protein. Clinical studies have shown that treated patients can experience significant improvements in motor function and survival outcomes compared with historical data from untreated SMA populations.

The UAE’s approval follows earlier regulatory authorization in another jurisdiction, underscoring growing international confidence in gene therapy approaches for rare genetic diseases. Health authorities in the region stated that the decision reflects the country’s strategy to accelerate patient access to innovative therapies and strengthen its position as a regional hub for advanced medical treatment.

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Industry observers note that regulatory approvals outside the U.S. and Europe are becoming increasingly important for the global commercialization of gene therapies. Faster adoption in regions such as the Middle East may help expand real-world evidence, support long-term safety monitoring, and improve access for patients who previously had limited treatment options.

As more countries evaluate similar therapies, the approval of Itvisma in the UAE highlights the broader trend of gene therapies transitioning from experimental interventions to clinically established treatment options worldwide.

Source: Times of India, UAE becomes second country to approve gene therapy Itvisma for spinal muscular atrophy, December 2025.