Itvisma SMA Gene Therapy Milestone Reinforces Abu Dhabi As A Global Healthcare Leader

Abu Dhabi has delivered the world’s first ITVISMA dose for spinal muscular atrophy at Sheikh Khalifa Medical City under the supervision of the Department of Health – Abu Dhabi. The one-time gene therapy, developed by Novartis, is administered to a patient with a confirmed SMN1 gene mutation, strengthening the emirate’s position in advanced genomic healthcare.

The UAE granted accelerated approval for ITVISMA on 25th November 2025, shortly after authorisation in the USA. With this decision, the country ranks among the earliest adopters of this SMA gene therapy worldwide. The move highlights Abu Dhabi’s efforts to expand access to high-level treatment options for rare neuromuscular diseases.

ITVISMA is designed as a single-dose gene therapy for patients aged 2 years and older who have a confirmed SMN1 gene mutation. The treatment replaces the missing or faulty SMN1 gene. This aims to improve motor function and reduce dependence on repeated therapies that many SMA patients would otherwise require over their lifetime.

According to officials, ITVISMA is intended to combine a straightforward dosing approach with long-term clinical effect. By delivering a one-time infusion, the therapy targets the root genetic cause of spinal muscular atrophy rather than only managing symptoms. This strategy aligns with precision medicine plans under development in Abu Dhabi’s healthcare sector.

Sheikh Khalifa Medical City, part of SEHA and a subsidiary of PureHealth, carried out the treatment under the guidance of the Department of Health – Abu Dhabi, which regulates the emirate’s healthcare sector. The procedure reflects growing collaboration between government bodies, healthcare providers and industry partners for genomic and gene-based care.

Dr. Noura Khamis Al Ghaithi, Under-Secretary of DoH, 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."

ITVISMA spinal muscular atrophy gene therapy Abu Dhabi regional role

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." The statement emphasised the importance of structured care pathways and close coordination between institutions.

Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, added, "Today’s 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 for advanced neuromuscular care." The collaboration is expected to support broader availability of gene therapies in the region.

The first administration of ITVISMA in Abu Dhabi combines early regulatory approval, clinical capability and industry cooperation in one project. Officials state that this development strengthens the emirate’s appeal for international patients, particularly those with rare conditions such as spinal muscular atrophy, while also setting a benchmark for future genomic and precision medicine initiatives in the Middle East.

With inputs from WAM