Abu Dhabi has achieved a historic global healthcare milestone by becoming the first location worldwide to administer ITVISMA™ (onasemnogene abeparvovec), a breakthrough gene replacement therapy for the treatment of spinal muscular atrophy (SMA). The achievement reinforces the emirate’s position as a leading global healthcare destination and highlights its commitment to genomics-driven, precision medicine.
Under the supervision of the Department of Health – Abu Dhabi (DoH), the regulator of the emirate’s healthcare sector, Sheikh Khalifa Medical City (SKMC)—part of SEHA, a subsidiary of PureHealth—successfully administered the innovative gene therapy developed by Novartis. ITVISMA™ is a one-time gene replacement therapy designed to address the root genetic cause of SMA in patients aged two years and older with a confirmed SMN1 gene mutation.
The therapy received accelerated regulatory approval in the UAE on November 25, 2025, positioning the country among the first globally—after the United States—to endorse this pioneering treatment. The milestone underscores Abu Dhabi’s strategic focus on rapidly translating cutting-edge medical innovation into patient access. Unlike conventional SMA treatments that require repeated dosing, ITVISMA™ works by replacing the missing or non-functional SMN1 gene, offering long-term therapeutic benefits and improved motor function.
H.E. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said the achievement reflects the emirate’s leadership in advanced healthcare delivery.
“This milestone demonstrates Abu Dhabi’s commitment to delivering world-class care and strengthening its position as a global leader in genomics and precision medicine. By administering ITVISMA™, we are proud to be among the first worldwide to offer this innovative treatment. Our priority remains safeguarding community health, ensuring access to life-changing therapies for rare diseases, and reinforcing Abu Dhabi’s standing as a leading destination for medical tourism.”
Bader Al Qubaisi, Chief Executive Officer at SKMC, highlighted the strength of Abu Dhabi’s integrated healthcare ecosystem.
“Delivering the world’s first ITVISMA™ treatment at SKMC is a testament to the seamless collaboration enabled by the Department of Health – Abu Dhabi. In alignment with PureHealth’s vision, coordinated care pathways and strategic partnerships—including with Novartis—have allowed us to bring this life-changing therapy to patients safely and efficiently.”
Emphasising the patient-centric impact, Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, said:
“This milestone is ultimately about patients and families. 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 centre for advanced neuromuscular care.”
Abu Dhabi’s approach to rare genetic diseases such as SMA is built on a comprehensive and integrated care model, encompassing universal screening, rapid confirmatory testing, timely referrals to DoH-designated specialists and centres of excellence, and early access to advanced therapies. The Department of Health plays a critical role through rapid health technology assessments, expedited product coding for coverage, and streamlined access pathways.
Further strengthening this ecosystem, Abu Dhabi’s Newborn Genetic Screening Programme offers whole genome sequencing to screen for over 815 treatable childhood genetic conditions, making it one of the most comprehensive newborn genetic screening programmes globally.
Source & Credits: Abu Dhabi Media Office
