Prime Highlights:
- The UAE has approved Itvisma, a gene therapy for SMA, for adults and children aged two and above, offering new hope to patients.
- The approval reinforces the UAE’s leadership in fast-tracking access to advanced medical treatments and rare disease therapies.
Key Facts:
- Itvisma works by replacing the faulty genethat causes SMA, improving motor function and reducing the need for long-term treatments.
- The Emirates Drug Establishment (EDE) approved itafter clinical evidence showed clear, sustained improvements and a positive safety profile.
Background:
The United Arab Emirates has taken a major leap in healthcare innovation by approving Itvisma (onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy (SMA). The treatment is now available for anyone aged two and above, making the UAE the second country to approve it.
The Emirates Drug Establishment (EDE) approved it after reviewing evidence showing clear improvements and a good safety record.
Dr. Fatima Al Kaabi, Director General of EDE, said the approval shows the UAE’s commitment to providing advanced genetic therapies, especially for rare diseases. She noted that the country’s regulatory system balances speed, transparency, and safety, so that new treatments reach patients quickly and reliably.
For Novartis, the maker of Itvisma, the approval is an important step in patient care and neuromuscular research. Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, said that working closely with EDE will allow patients who previously couldn’t access one-time gene therapy to get it faster and fairly.
He added that the UAE’s support for advanced treatments can improve the course of SMA and fits with Novartis’s focus on neurology and neurodegenerative diseases.
The approval also shows that the UAE is becoming a regional center for pharmaceutical innovation and is committed to improving healthcare for patients in the region.
