The United Arab Emirates has approved the use of an advanced gene therapy to treat adults and children diagnosed with spinal muscular atrophy (SMA). This decision marks a significant step toward providing cutting-edge treatments for patients with rare genetic diseases across Dubai and the UAE.
The drug, Itevisma, developed by the Swiss pharmaceutical company Novartis, works by replacing the defective SMN1 gene responsible for this debilitating condition. Administered as a single fixed dose, the therapy aims to improve patients’ quality of life and reduce dependence on long-term treatments.
The UAE’s drug regulatory authority granted approval following clinical trials that demonstrated sustained improvements in patients’ motor functions. The UAE is now the second country to authorize the use of Itevisma for adults and children aged two and older, after its approval by the U.S. Food and Drug Administration (FDA) in November.
What is Spinal Muscular Atrophy?
Spinal muscular atrophy affects motor neurons—the nerve cells responsible for sending signals to muscles—leading to muscle weakness. Some patients are unable to walk or experience significant difficulty with movement, while the disease can also impair swallowing and breathing.
Children with the most severe form of SMA, who are unable to sit without support, typically do not survive beyond the age of two. Symptoms of this form usually appear within the first six months of life. Other types of SMA affect older children and adults, presenting milder yet still impactful symptoms.
Rapid Access to Advanced Treatments in the UAE
The approval of Itevisma highlights the UAE’s commitment to providing state-of-the-art genetic therapies, particularly for patients with rare genetic disorders such as SMA. It also reflects the efficiency of the country’s healthcare system in evaluating and approving innovative medicines.
Treatments for spinal muscular atrophy are extremely costly, limiting access for many patients. Itevisma delivers a functional SMN gene to cells, enabling the production of the essential SMN protein. As a one-time treatment with a cost of approximately one million dirhams, it is considered one of the most expensive drugs in the world.
