The Emirates Drug Establishment has approved Itvisma (onasemnogene abeparvovec), a gene therapy for the treatment of spinal muscular atrophy (SMA) in eligible adults and children aged two and above.
Itvisma addresses the genetic root of SMA by replacing the faulty gene responsible for the disease, helping improve motor function and reduce dependence on long-term treatments © Mena Today
The Emirates Drug Establishment has approved Itvisma (onasemnogene abeparvovec), a gene therapy for the treatment of spinal muscular atrophy (SMA) in eligible adults and children aged two and above.
With this decision, the United Arab Emirates becomes the second country worldwide to authorise the therapy, underscoring its drive to accelerate patient access to advanced medical innovations.
Itvisma addresses the genetic root of SMA by replacing the faulty gene responsible for the disease, helping improve motor function and reduce dependence on long-term treatments.
Dr Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, said the approval reflects the UAE’s commitment to providing patients with cutting-edge genetic therapies, particularly for rare diseases. She noted that the decision was based on strong clinical evidence showing sustained motor improvements and a positive safety profile.
Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, said the approval marks a major step for patients and families living with SMA, offering new access to a one-time gene therapy through close cooperation with UAE health authorities.
The approval supports the UAE’s broader goal of building a world-class, innovative, and sustainable healthcare system while positioning the country as a regional hub for pharmaceutical innovation.
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