A leading Abu Dhabi hospital has become the first in the world to deliver a pioneering gene-therapy treatment that is poised to offer a lifeline to children and adults with spinal muscular atrophy.
Sheikh Khalifa Medical City successfully administered Itvisma, a one-dose drug which works to replace the defective SMN1 gene responsible for the debilitating disease, under the supervision of the emirate’s Department of Health.
Authorities did not reveal any details about the patient who received the treatment or when the procedure took place. Nor did they reveal its price tag, although it is expected to be expensive as it works in a similar way to Zolgensma, which costs around $2 million per patient.
The drug, developed by Swiss pharmaceutical company Novartis, was given accelerated approval by the UAE on November 25, following clinical trials that demonstrated sustained improvements in patients’ motor abilities.
It was only the second country − after the US − to approve the use of Itvisma, which targets children aged two and above, as well adults with SMA.
For several years, doctors in the UAE have successfully been administering another gene-therapy drug, Zolgensma, to children with SMA. However, this medicine is suitable only for infants.
SMA can cause severe disability and, in some cases, proves fatal in young children.
“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,” said Dr Noura Khamis Al Ghaithi, undersecretary of the Department of Health on Sunday.
“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 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.”
Mohamed Ezz Eldin, head of the GCC Cluster at Novartis, highlighted the significance of the milestone medical achievement for patients.
“Today’s milestone is ultimately about patients and families,” he 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.”
SMA affects motor neurons, which are nerve cells that send impulses to the muscles, and causes limb weakness.
Some people with the condition are unable or find it difficult to walk. SMA may also make swallowing and breathing difficult.
Children who have the most severe form, Type 1, and who are not treated cannot sit unsupported and typically die before their second birthday, according to the US National Institutes of Health.
Growing support network
The first treatment in the Emirates comes after Medcare Royal Specialty Hospital in Dubai’s Al Qusais this week announced it was poised to become one of the first centres outside of the US to offer the SMA drug.
Clinicians at the centre said they expect that patients will travel to Dubai from across the globe to receive the drug.