UAE Approves Gene Therapy ‘Itvisma’ to Treat Children with Spinal Muscular Atrophy

The UAE has approved the use of Itvisma, a groundbreaking gene therapy developed by Novartis, to treat adults and children with spinal muscular atrophy (SMA). The therapy replaces the defective SMN1 gene responsible for the disease, aiming to improve patients’ motor function, quality of life, and reduce dependence on long-term treatments.
Itvisma, delivered in a single dose, is only the second country-approved treatment after the US FDA in November. Clinical trials demonstrated significant improvements in patients’ motor abilities, prompting the Emirates Drug Establishment to grant regulatory approval for children aged two and above and adults. SMA is a genetic condition that affects motor neurons, causing limb weakness, difficulty in walking, and in severe cases, challenges in breathing and swallowing.
Officials highlighted that the approval reflects the UAE’s commitment to providing access to advanced genetic therapies and rare disease treatments. Itvisma, a more advanced version of Novartis’ Zolgensma, is known as the world’s most expensive drug, costing around $2 million per single-dose treatment. While the availability date and pricing in the UAE remain undisclosed, the therapy offers hope to SMA patients and their families, with previous cases in the UAE demonstrating rapid access and successful outcomes.