UAE approves gene replacement therapy to treat spinal muscular atrophy

The UAE has become the second country in the world to approve the use of Itvisma to treat spinal muscular atrophy (SMA) in eligible adults and children aged two years and above.

Making the announcement, the Emirates Drug Establishment (EDE) explained that Itvisma (onasemnogene abeparvovec) is a gene therapy that uses an adeno-associated viral vector.

It targets the genetic cause of spinal muscular atrophy by replacing the defective gene responsible for the disease, helping to improve patients’ motor functions and reduce their reliance on long-term treatments.

Dr. Fatima Al Kaabi, Director-General of the Emirates Drug Establishment, said it's a "significant step demonstrating the UAE's commitment to providing patients with the latest advanced genetic therapies, particularly those with rare genetic diseases like spinal muscular atrophy".

“This decision showcases the efficiency of the country's health system and its capability to evaluate and approve innovative drugs according to the highest scientific standards within effective timeframes."

She clarified that Itvisma was approved following clinical evidence that resulted in clear and sustained improvements in patients’ motor abilities, in addition to a consistently positive safety profile observed throughout all stages of clinical evaluation.