Abu Dhabi uses breakthrough gene therapy treatment for spinal muscular atrophy

Abu Dhabi became one of the first cities in the world to use ITVISMA (onasemnogene abeparvovec), the breakthrough gene therapy treatment developed by Novartis for spinal muscular atrophy (SMA).

ITVISMA is a one-time gene therapy specifically designed to target the underlying genetic cause of SMA in patients aged two years and older with a confirmed SMN1 gene mutation. ITVISMA replaces the missing SMN1 gene to improve motor function, reducing the need for continuous treatments required by other therapies.

Sheikh Khalifa Medical City (SKMC), part of SEHA and a subsidiary of PureHealth, successfully administered the treatment under the supervision of the Department of Health – Abu Dhabi (DoH).

World’s first ITVISMA SMA treatment

Abu Dhabi thus became the first city in the world to deliver the treatment, reinforcing its status as a leading global healthcare destination and demonstrating its commitment to patient care through genomics and precision medicine.

The groundbreaking therapy received accelerated approval in the UAE on 25 November this year, a day after it was approved by the US Food & Drug Administration (FDA). This positioned the UAE among the first countries globally, following the USA, to endorse this pioneering treatment.

At the time of the FDA approval, Novartis said: “The approval of ITVISMA is based on data from the registrational Phase lll STEER study and supported by the open-label Phase lllb STRENGTH study.

“Itvisma showed statistically significant improvements in motor function and stabilisation of motor abilities typically not seen in the natural history of the disease, with effects sustained over 52 weeks of follow-up.

“Additionally, ITVISMA demonstrated a safety profile with adverse events that were consistent across both studies. The most common adverse events in the STEER study were upper respiratory tract infection and pyrexia, and the most common adverse events in the STRENGTH study were common cold, pyrexia, and vomiting.”

Dr Noura Khamis Al Ghaithi, Under-Secretary of DoH, said: “This milestone reflects Abu Dhabi’s commitment to delivering world-class 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.”

Bader Al Qubaisi, Chief Executive Officer at SKMC, added: “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.”

Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, said: “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.”