Abu Dhabi Delivers World’s First ITVISMA Gene Therapy

Abu Dhabi has achieved a historic milestone by becoming the first city worldwide to administer ITVISMA (onasemnogene abeparvovec) gene therapy for spinal muscular atrophy (SMA). This landmark step highlights the emirate’s leadership in genomics, precision medicine, and advanced healthcare solutions.

Under the supervision of the Department of Health – Abu Dhabi (DoH), SEHA Sheikh Khalifa Medical City (SKMC), part of SEHA and a subsidiary of PureHealth, successfully delivered the therapy developed by Novartis. ITVISMA is a one-time gene therapy designed to target the root genetic cause of SMA in patients aged two years and older with a confirmed SMN1 gene mutation.

The UAE granted accelerated approval for ITVISMA on 25 November 2025, making the country one of the first globally, after the US, to authorize this groundbreaking treatment. Unlike other therapies that require ongoing administration, ITVISMA replaces the missing SMN1 gene, improving motor function and offering long-term benefits for patients.

Key Statements:

• Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, stated:
  "This milestone demonstrates Abu Dhabi’s commitment to world-class care and innovative healthcare. Administering ITVISMA reinforces our position as a global leader in genomics and precision medicine, ensuring patients access life-changing therapies for rare diseases."

• Bader Al Qubaisi, CEO of SKMC, said:
  "Delivering the world’s first ITVISMA treatment reflects Abu Dhabi’s integrated healthcare ecosystem. Collaboration with partners such as Novartis enables us to provide safe and efficient access to this transformative therapy."

• Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, commented:
  "This achievement is about patients and families. Working with DoH and SKMC, we are proud to expand access to breakthrough treatments like ITVISMA, strengthening Abu Dhabi’s role in advanced neuromuscular care."

Comprehensive SMA Care in Abu Dhabi:

Abu Dhabi’s approach to rare genetic diseases, including SMA, involves:

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

• Rapid confirmatory testing and timely referrals to DoH-designated specialists and centers of excellence.

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

This integrated model positions Abu Dhabi as a global reference for rare disease care, combining innovation, precision medicine, and patient-centered treatment pathways.