In a landmark decision for medical advancement, the United Arab Emirates has significantly broadened access to a groundbreaking gene therapy for spinal muscular atrophy (SMA). The Emirates Drug Establishment (EDE) has granted regulatory approval for Itvisma (onasemnogene abeparvovec) to now include eligible adults and children aged two years and above.
This authorization elevates the UAE to the position of the second nation worldwide to approve this innovative treatment, solidifying its status as a regional pioneer in facilitating patient access to cutting-edge medical solutions. The therapy utilizes an adeno-associated viral vector to directly address the genetic root cause of SMA by delivering a functional replacement for the defective SMN1 gene.
Dr. Fatima Al Kaabi, Director-General of the EDE, emphasized the decision’s significance, stating it underscores the UAE’s dedication to integrating advanced genetic therapies into its healthcare framework, particularly for rare genetic disorders. The approval followed rigorous evaluation of clinical evidence demonstrating sustained improvements in patients’ motor functions and a consistently positive safety profile throughout all trial phases.
The regulatory move is a strategic component of the UAE’s vision to cultivate a world-class, innovative, and sustainable healthcare ecosystem. Dr. Al Kaabi highlighted the efficiency of the national health system in evaluating and approving novel pharmaceuticals according to the highest international scientific standards, while maintaining a careful balance between speed, scientific rigor, and transparency.
Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis—the therapy’s developer—hailed the approval as a pivotal achievement for patients and their families. He noted that close collaboration with the EDE aims to ensure rapid and equitable access to this one-time interventional treatment, potentially altering the disease’s trajectory for many.
This milestone also reflects Novartis’s expanding commitment to neurology, building on its expertise in SMA and multiple sclerosis to address neuroimmunology, neurodegenerative, and neuromuscular diseases. For the UAE, it strategically reinforces its ambition to become a regional hub for pharmaceutical innovation, delivering high-value therapies that meet stringent quality and safety benchmarks.