Abu Dhabi Unveils World’s First ITVISMA Gene Therapy for SMA

Post by : Bianca Suleiman

Abu Dhabi has marked a momentous achievement by being the first city globally to administer ITVISMA (onasemnogene abeparvovec) gene therapy for spinal muscular atrophy (SMA). This groundbreaking move underscores the emirate’s prowess in genomics, precision medicine, and cutting-edge healthcare solutions.

Supervised by the Department of Health – Abu Dhabi (DoH), SEHA Sheikh Khalifa Medical City (SKMC), which is part of SEHA and operates under PureHealth, has successfully administered the therapy developed by Novartis. ITVISMA is a one-off gene therapy that targets the fundamental genetic issue of SMA in individuals aged two years and above with a verified SMN1 gene mutation.

On November 25, 2025, the UAE granted expedited approval for ITVISMA, positioning the country as one of the earliest adopters worldwide, following the US, in sanctioning this revolutionary treatment. Unlike alternative therapies that necessitate continuous administration, ITVISMA substitutes the absent SMN1 gene, enhancing motor function and providing enduring advantages for patients.

Notable Quotes:

• Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, remarked:
  "This landmark reflects Abu Dhabi’s dedication to top-tier healthcare and innovation. The administration of ITVISMA strengthens our stance as a global forerunner in genomics and precision medicine, ensuring that patients have access to life-altering therapies for rare disorders."

• Bader Al Qubaisi, CEO of SKMC, expressed:
  "The rollout of the world’s first ITVISMA treatment showcases Abu Dhabi’s cohesive healthcare framework. Partnering with entities like Novartis enables us to offer safe and efficient access to this transformative treatment."

• Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, commented:
  "This remarkable milestone centers on the well-being of patients and their families. Collaborating with DoH and SKMC, we are proud to broaden access to pioneering treatments like ITVISMA, further solidifying Abu Dhabi’s significance in advanced neuromuscular care."

Holistic SMA Management in Abu Dhabi:

Abu Dhabi’s strategy for tackling rare genetic disorders like SMA incorporates:

• Universal newborn screening and comprehensive genome sequencing for over 815 manageable genetic conditions.

• Prompt confirmatory testing and swift referrals to DoH-certified specialists and centers of excellence.

• Accelerated access to early therapies through streamlined health technology assessments and coverage approvals.

This integrated framework positions Abu Dhabi as a worldwide benchmark for rare disease treatment, merging innovation, precision medicine, and patient-oriented care pathways.