The UAE has granted regulatory approval for a new gene therapy designed to treat spinal muscular atrophy (SMA), a rare genetic disorder that affects muscle strength and motor function. The approval was issued by the Emirates Drug Establishment, marking a significant step in expanding access to advanced genetic treatments within the country.
The therapy, developed by Novartis, works by replacing the defective SMN1 gene responsible for SMA. Administered as a single-dose infusion, the treatment aims to improve motor development and reduce long-term dependence on supportive care.
The UAE becomes one of the early adopters globally to authorise this newer formulation, following earlier regulatory clearance by the U.S. Food and Drug Administration (FDA).
Understanding Spinal Muscular Atrophy
According to the U.S. National Institutes of Health (NIH), spinal muscular atrophy is a genetic condition that damages motor neurons — the nerve cells that control voluntary muscle movement. The disease can impair walking, swallowing, and breathing.
The most severe form, Type 1 SMA, typically appears within the first six months of life and, without treatment, can significantly reduce life expectancy. Other forms (Types 2, 3, and 4) present with varying levels of severity but may still result in progressive muscle weakness.
The Cost and Context of Gene Therapy
Earlier versions of gene therapy for SMA — including Novartis’ Zolgensma — have been widely reported as among the world’s most expensive single-dose treatments, with pricing previously reaching approximately $2 million per patient. Details about Zolgensma are available through Novartis’ official product information.
While pricing and availability timelines for the newly approved therapy in the UAE have not yet been publicly confirmed, health authorities emphasised that the decision reflects the country’s commitment to rare disease treatment and precision medicine.
Dr Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, stated that the approval highlights the UAE’s ability to evaluate innovative therapies according to rigorous scientific standards while accelerating patient access.
A Broader Commitment to Rare Disease Care
The UAE has increasingly prioritised advanced therapies for rare genetic disorders. Dubai Health’s Al Jalila Children’s Hospital has previously administered gene therapy treatments for SMA patients, reinforcing the country’s infrastructure for specialised paediatric care.
Health regulators continue to monitor clinical outcomes and global research developments to ensure safety and efficacy standards are upheld.
As gene therapy evolves, its long-term impact will depend not only on regulatory support but also on accessibility, funding frameworks, and integration into national healthcare systems.
Sources
• Emirates Drug Establishment
• Novartis Global
• U.S. Food and Drug Administration (FDA)
• U.S. National Institutes of Health (NIH)
Aisha Al Mansoori is a UAE-based business and technology analyst covering startups, venture capital, AI, fintech, and innovation trends shaping the Emirates’ economy.
