Abu Dhabi Sets a Global Precedent with ITVISMA Gene Therapy

Abu Dhabi has made history as the first city in the world to administer ITVISMA (onasemnogene abeparvovec) gene therapy for spinal muscular atrophy (SMA). This significant achievement underlines the emirate's leadership in genomics, cutting-edge medicine, and advanced health solutions.

Supervised by the Department of Health – Abu Dhabi (DoH), SEHA Sheikh Khalifa Medical City (SKMC), part of SEHA and a subsidiary of PureHealth, successfully delivered this therapy created by Novartis. ITVISMA is a one-off gene therapy aimed at addressing the genetic root of SMA in patients aged two years and older who possess a confirmed SMN1 gene mutation.

The UAE granted accelerated approval for ITVISMA on November 25, 2025, positioning the nation among the first globally to endorse this innovative treatment after the United States. Unlike conventional therapies that necessitate ongoing administration, ITVISMA substitutes the absent SMN1 gene, enhancing motor function and conferring long-lasting benefits for patients.

Key Statements:

• Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, noted:
  "This achievement signifies Abu Dhabi's dedication to exceptional healthcare and innovative medical practices. The administration of ITVISMA solidifies our standing as a world leader in genomics and precision medicine, ensuring that patients can access transformative therapies for rare diseases."

• Bader Al Qubaisi, CEO of SKMC, remarked:
  "Providing the first ITVISMA treatment in the world showcases Abu Dhabi’s comprehensive healthcare ecosystem. Partnerships with entities like Novartis enable us to ensure safe and effective access to this groundbreaking therapy."

• Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, expressed:
  "This milestone is centered on patients and their families. Through collaboration with DoH and SKMC, we are excited to enhance access to pioneering treatments such as ITVISMA, fortifying Abu Dhabi’s commitment to advanced neuromuscular care."

Comprehensive SMA Care in Abu Dhabi:

Abu Dhabi’s strategy for managing rare genetic diseases, including SMA, encompasses:

• Universal newborn screenings and whole genome sequencing for over 815 treatable genetic conditions.

• Swift confirmatory testing and prompt referrals to DoH-designated specialists and centers of excellence.

• Quick access to early interventions through streamlined health technology evaluations and coverage authorizations.

This integrated approach establishes Abu Dhabi as a global benchmark for rare disease treatment, merging innovation, precision medicine, and patient-focused care pathways.