Post by : Badri Ariffin

Abu Dhabi has marked a significant milestone by being the first city anywhere in the world to administer ITVISMA (onasemnogene abeparvovec) gene therapy targeting spinal muscular atrophy (SMA). This pioneering move showcases the emirate’s leadership in genomics, precision medicine, and healthcare innovation.

Under the guidance of the Department of Health – Abu Dhabi (DoH), the SEHA Sheikh Khalifa Medical City (SKMC), part of SEHA and affiliated with PureHealth, successfully delivered this therapy developed by Novartis. ITVISMA is a single-dose gene therapy designed to address the underlying genetic cause of SMA in patients aged two and older with a verified SMN1 gene mutation.

The UAE granted expedited approval for ITVISMA on November 25, 2025, making it one of the first countries, following the US, to endorse this revolutionary treatment. Unlike conventional therapies that necessitate continual administration, ITVISMA effectively replaces the absent SMN1 gene, bolstering motor function and offering extended benefits for patients.

Key Statements:

• Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, remarked:
  "This achievement underscores Abu Dhabi’s dedication to delivering world-class healthcare and innovation. The administration of ITVISMA solidifies our status as a global hub for genomics and precision medicine, ensuring adequate access to life-changing therapies for rare diseases."

• Bader Al Qubaisi, CEO of SKMC, stated:
  "Administering the first-ever ITVISMA treatment reflects Abu Dhabi’s cohesive healthcare ecosystem. Collaborations with companies like Novartis allow us to provide secure and effective access to transformative therapies."

• Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, commented:
  "This milestone emphasizes the needs of patients and families. Partnering with DoH and SKMC, we take pride in broadening access to groundbreaking treatments like ITVISMA, enhancing Abu Dhabi’s prominence in advanced neuromuscular care."

Comprehensive SMA Care in Abu Dhabi:

Abu Dhabi’s strategy for addressing rare genetic conditions such as SMA includes:

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

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

• Accelerated access to early therapies via effective health technology assessments and approval processes.

This integrated care model positions Abu Dhabi as a global leader in rare disease management, merging innovation, precision medicine, and patient-centric treatment pathways.

#Abu Dhabi News #UAE News