The United Arab Emirates has reached a major milestone in the field of medical research and treatment services by becoming the second country worldwide to greenlight Itvisma, the new gene therapy developed for the treatment of spinal muscular atrophy in patients aged two years and above.
On December 20, 2025, the Emirates Drug Establishment (EDE) announced the approval of the drug, emphasizing the UAE’s efforts to accelerate the adoption of state-of-the-art healthcare technologies capable of treating rare genetic disorders.
However, before EDE’s approval, the US FDA had greenlit the drug for release into the US market, thereby making the UAE a pioneer among nations in accepting innovations in healthcare technology beyond US borders.
Itvisma is a gene replacement therapy developed by the pharmaceutical giant Novartis. It can be used to treat spinal muscular atrophy by targeting the root cause: replacing a defective gene in a cell. The therapy can result in a considerable improvement in motor function in patients and reduce their lifelong reliance on a particular course of treatment.
Contrary to other treatments that need frequent dosage, Itvisma is only taken once, providing a revolutionary therapy for people suffering from SMA. Clinical trial efficacy demonstrated improvements in motor function and good tolerability, which were essential to the UAE’s approval of the treatment.
Dr Fatima Al Kaabi, Director General of the EDE, said, “This approval is a reflection of the UAE's commitment to giving patients access to the most up-to-date, most advanced genetic therapies, and, in particular, those with rare genetic conditions such as spinal muscular atrophy.”
Al Kaabi praised the efficiency of the UAE's regulatory bodies in approving new treatments within a science-supported timeframe. Mohamed Ezz Eldin, Head of the GCC Cluster of Novartis, noted that the approval will also enable the fast and equitable distribution of this life-changing medicine to patients and families who had limited prior options.
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Its approval supports the UAE's aim to emerge as an innovative hub in pharmaceuticals. This fits well with the UAE's vision of becoming an icon of a world-class, efficient, and sustainable healthcare system. There is optimism about the UAE regulatory regime, which strikes the right balance between scientific standards, speed, and transparency.