The Emirates Drug Authority (EDA) has announced the approval of Itvisma, a gene therapy based on an adenovirus vector, for the treatment of spinal muscular atrophy (SMA) in adults and children aged two years and older who are medically eligible. With this approval, the UAE becomes the second country globally to grant this treatment, reinforcing its leadership in accelerating access to advanced medical innovations for patients in the region.

Itvisma works by targeting the genetic cause of SMA through gene replacement, thereby improving patients’ motor functions and reducing their reliance on long-term treatments.

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This accreditation comes as part of the Emirates Drug Establishment’s ongoing efforts to provide advanced and high-value treatments, and to accelerate access to pharmaceutical innovations for the Emirati community, in line with the country’s direction towards developing a leading health sector based on efficiency, innovation and sustainability.

Her Excellency Dr. Fatima Al Kaabi, Director General of the Emirates Drug Authority, stated:
“The regulatory approval of Itvisma is a pivotal step reflecting the UAE’s commitment to providing patients with the latest advanced gene therapies, particularly those suffering from rare genetic diseases such as spinal muscular atrophy. This decision demonstrates the efficiency of the UAE’s healthcare system and its ability to evaluate and approve innovative medicines according to the highest scientific standards and within effective timeframes, ensuring patients have rapid access to leading treatment options that enhance their quality of life.”

Dr. Al Kaabi explained that the approval of Itvisma is based on the results of clinical studies that demonstrated a clear improvement in patients’ motor abilities and the long-term sustainability of these benefits, in addition to the treatment’s positive safety profile during the clinical evaluation phases.

She added: “The UAE’s granting of regulatory approval for this treatment reflects its leadership in adopting and implementing advanced pharmaceutical technologies and reinforces international confidence in the ability of its regulatory model to achieve a precise balance between speed, accuracy, and transparency, ensuring that innovative treatments reach patients in a timely manner.”

She concluded: “The Emirates Drug Authority will continue to support medical innovation and the adoption of modern treatments that improve quality of life, in line with the UAE’s vision to build a leading healthcare sector based on knowledge, efficiency, and sustainability.”

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Mohammed Ezzeldin, Regional Director of Novartis in the GCC region, also emphasized that the approval of Etvisma in the UAE is primarily for the benefit of patients and their families. He noted that close collaboration with the Emirates Drug Authority and partners aims to ensure rapid and equitable access to treatment, giving individuals with spinal muscular atrophy (SMA) who were previously unable to benefit from single-dose gene therapy a new opportunity to access this type of medical intervention.

He added that the UAE’s support for accelerating the approval of innovative treatments contributes to changing the course of SMA, affirming the company’s pride in its role in this regard. Medical progress. He noted that this achievement reflects Novartis’ commitment to the field of neurology, based on its extensive experience in treating spinal muscular atrophy and multiple sclerosis, and in conjunction with its expansion into the areas of neuroimmunology, neurodegenerative disorders, and neuromuscular diseases.

The regulatory approval of Etvisma comes within the framework of the Emirates Drug Authority’s strategy to enhance the UAE’s position as a regional hub for pharmaceutical innovation by providing advanced gene therapies of the highest quality and safety standards. The Authority employs a modern regulatory model based on rigorous governance, transparency, and global partnerships to support research and development, adopt cutting-edge technologies, and provide integrated care.

This approach contributes to ensuring patients’ efficient access to advanced medicines and strengthens the country’s readiness in the healthcare sector, in line with the UAE’s vision of building a leading and sustainable healthcare system.