A Dubai hospital has announced it will become one of the first centres outside of the US to offer a groundbreaking new gene-therapy treatment for spinal muscular atrophy.
Medcare Royal Specialty Hospital in Al Qusais will provide the newly licensed treatment, called Itvisma, to patients with spinal muscular atrophy or SMA who are aged above two years old or weigh more than 21kg.
Clinicians at the centre said they expect that patients will travel to Dubai from across the globe to receive the drug.
Zolgensma, a gene-therapy treatment for SMA that was licensed several years ago, was available only to very young children, limiting treatment options for older people with the disease.
SMA can cause severe disability and, in some cases, proves fatal in young children.
Expanding support
Earlier this month the Emirates Drug Establishment (EDE) granted regulatory approval for Itvisma to be made available in the UAE, making the country just the second in the world to give the drug the green light. The US Food and Drug Administration approved the treatment last month.
Patients who live in the UAE, the region and further afield are likely to be treated with Itvisma at Medcare Royal Specialty Hospital.
Since 2020, Medcare, a private healthcare provider that is part of Aster DM Healthcare, has used gene therapy to treat 180 children with SMA, among them patients who have travelled from Iran, Turkey, Nepal, Romania, India and Ethiopia.
While the cost of Itvisma has not been publicly announced, it is expected to carry a heavy price tag given that Zolgensma, which works in a similar way, has been described as the world’s most expensive medicine, costing around $2 million per patient.
Transforming care
Dr Vivek Mundada, a consultant paediatric neurologist at Medcare Royal Specialty Hospital, said that the new drug offered “a paradigm shift” by allowing older and heavier children, and adults, to be treated.
It was likely, he said, to help them to stand, move their arms and breathe more comfortably than would otherwise have been the case.
Based on the experience with Zolgensma, he is “very optimistic” that the UAE authorities will provide funding for Emirati patients to be treated with Itvisma, which was developed by the pharmaceutical giant Novartis.
While saying that approval from the EDE was the first step to making the UAE “a global hub” for the treatment, he said that ensuring that UAE national families were at the forefront of access was “a top priority”.
“The UAE has a proud history of visionary leadership in healthcare,” he said. “For Zolgensma, the authorities and government-backed programmes were instrumental in ensuring Emirati families had access to treatment.
“Given that the UAE is now the first country outside the US to approve this new intrathecal [administered to the spinal theca] therapy for older and heavier patients, we anticipate a similar level of support.”
Other options, likely to be more relevant to families from other nationalities being treated in the UAE, include medical insurance and private or community funding.
“For families with high-tier insurance coverage, we are working to ensure the clinical necessity of this one-time gene therapy is recognised,” Dr Mundada said.
“However, because gene therapy represents a significant frontier in medicine, many families currently rely on the incredible generosity of the community.
“We see a significant number of families successfully utilising crowdfunding and the support of registered UAE charities to raise the necessary funds.”
Dr Mundada said that he provided clinical documentation and support for families working to secure funding.
While there is no formal patient support programme for the new therapy, Dr Mundada said that Medcare was building on a model developed by institutions such as Al Jalila Children’s Hospital in Dubai, which has also been central to the treatment of SMA in the UAE.
Debilitating condition
Most cases of SMA are the result of a mutation in a gene called survival motor neuron 1, or SMN1, which codes for the survival motor neuron protein.
If the body cannot produce this protein normally, the activity of motor neurons – nerve cells carrying signals from the brain to the muscles – is badly affected, causing weakness in the limbs and other problems, such as difficulty breathing or swallowing.
There are four main types of the condition, from 1 to 4, with type 1 affecting children earliest and having the most severe effects, with death at a young age likely without treatment.
Itvisma and Zolgensma each provide a normal form of the SMN1 gene so that the patient’s cells can produce the survival motor neuron protein.
Other SMA treatments require repeated spinal injections, but because it addresses the genetic cause of the disease, the new therapy only has to be applied once only.
Dr Shanila Laiju, group CEO of Medcare Hospitals and Medical Centres, described the availability of the new treatment as “a defining moment for SMA care in the region and beyond”.
“For families who have exhausted options or been told their child does not meet the criteria for gene therapy, this development offers something incredibly powerful – a renewed opportunity for improved quality of life and functional outcomes,” she said.
