Abu Dhabi Becomes the First in the World to Administer Breakthrough Gene Replacement Therapy (ITVISMA™) for Spinal Muscular Atrophy

28 Dec, 2025:

Abu Dhabi marks landmark achievement in becoming the first in the world to deliver ITVISMA™ (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA), reinforcing the emirates position a leading global healthcare destination and demonstrating its commitment to patient care through genomics and precision medicine.

Under the supervision of the Department of Health – Abu Dhabi, the regulator of the healthcare sector in the emirate, Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth, successfully administered this groundbreaking gene therapy developed by Novartis. ITVISMA™ is a one-time gene therapy specifically designed to target the underlying genetic cause of SMA in patients aged 2 years and older with a confirmed SMN1 gene mutation.

This groundbreaking therapy received accelerated approval in the UAE on November 25, 2025, positioning the UAE among the first countries globally – after the USA to endorse this pioneering treatment. This underscores Abu Dhabi's commitment to bringing cutting-edge innovations to patients across the region. Designed for simplicity long-term impact, ITVISMA™ replaces the missing SMN1 gene to improve motor function, reducing the need for continuous treatments required by other therapies.

H.E. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said: “This milestone reflects Abu Dhabi’s commitment to delivering worldclass care and strengthening its position as a global leader in healthcare driven by genomics and precision medicine. By administering ITVISMA™, we are proud to be among the first to provide this innovative treatment, further reinforcing our role as a leader and accelerator in advanced and innovative healthcare. Our priority remains safeguarding the health of our community members and beyond ensuring access to cutting-edge therapies for rare diseases, supporting the emirate’s standing as a leading destination for medical tourism. This achievement is just one of many as we remain dedicated to accelerating treatment to life-changing therapies and providing patients with access to the latest advancements in healthcare.”

Bader Al Qubaisi, Chief Executive Officer at SKMC, said: “Delivering the world’s first ITVISMA™ treatment at SKMC is a testament to Abu Dhabi’s integrated healthcare ecosystem under the leadership of the Department of Health – Abu Dhabi. . In line with PureHealth’s vision and DoH’s guidance in defining seamless care pathways, , and facilitating collaboration with partners such as Novartis has enabled us to bring this life-changing therapy to our patients safely and efficiently.”

“Today’s milestone is ultimately about patients and families,” said Mohamed Ezz Eldin, Head of GCC Cluster at Novartis. “By working closely with the Department of Health – Abu Dhabi, and SKMC, we are proud to support accelerated access to breakthrough therapies such as ITVISMA™, and to contribute to Abu Dhabi’s growing role as a regional and global reference for advanced neuromuscular care.”

In Abu Dhabi, care pathways for rare genetic diseases, such as SMA, are built on a comprehensive and integrated approach that encompasses universal screening, rapid confirmatory testing, timely referral to DoH-designated specialists and centers of excellence, and access to early therapy where indicated. DoH plays a critical role in conducting a rapid health technology assessment, expediting product coding for access and coverage purposes and ensuring the timely availability of innovative therapies to patients. The Newborn Genetic Screening Programme further strengthens this model by offering whole genome sequencing to screen over 815 treatable childhood genetic conditions, positioning it among the most comprehensive newborns genetic screening programmes in the world.