In a landmark move for rare disease care, the United Arab Emirates has approved Itvisma (onasemnogene abeparvovec), a cutting-edge gene therapy for spinal muscular atrophy (SMA). The therapy, developed by Swiss pharmaceutical company Novartis, is now available for children and adults aged two years and older, giving hope to patients and families who have long faced limited treatment options.
SMA is a genetic disorder that progressively weakens muscles, affecting movement, breathing, and swallowing. Traditional treatments manage symptoms, but Itvisma addresses the root cause by replacing the faulty SMN1 gene with a healthy copy. Administered as a single dose, the therapy has shown remarkable results in improving motor function and muscle strength in clinical studies.
Dr. Fatima Al Kaabi, Director-General of the Emirates Drug Establishment (EDE), said the approval marks an important step in expanding access to innovative therapies. “We are committed to providing patients with safe and timely access to life-changing treatments. Itvisma exemplifies how science can transform lives,” she said.
Novartis highlighted the UAE’s approval as part of a collaborative effort with healthcare authorities to ensure equitable access for patients. “For families living with SMA, this therapy offers more than treatment—it offers hope,” the company said.
The decision also reflects the UAE’s ambition to become a regional hub for advanced medical care. By enabling access to world-class therapies, the country is strengthening its healthcare ecosystem while giving patients a chance at a brighter, more independent future.
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