Emirates Drug Establishment approves gene replacement therapy for new age group in spinal muscular atrophy patients

• UAE becomes world's second country to approve "Itvisma" following US FDA.
• " Itvisma" is a single-dose, fixed genetic therapy for the treatment of spinal muscular atrophy.

Dubai, UAE, December 20, 2025

The Emirates Drug Establishment(EDE)  has announced the approval of Itvisma™ (onasemnogene abeparvovec), a gene therapy that uses an adeno-associated viral vector to treat spinal muscular atrophy (SMA) in eligible adults and children aged two years and above.

With this decision, the UAE becomes the second country globally to grant approval for this treatment, reinforcing its leadership in accelerating patient access to advanced medical innovations across the region.

Itvisma 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.

This regulatory authorisation is part of EDE’s ongoing commitment to enabling access to high-value, advanced therapies and accelerating the delivery of breakthrough medicines to UAE patients, in line with the UAE’s vision for a world-class, efficient, innovative, and sustainable healthcare ecosystem.

Her Excellency Dr. Fatima Al Kaabi, Director General of the Emirates Drug Establishment, stated: "Granting the regulatory approval for Itvisma is 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. We are committed to ensuring patients receive rapid access to cutting-edge treatment options that improve their quality of life,” Al Kaabi said.

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.

Al Kaabi added: " The UAE’s decision to grant regulatory approval for this therapy reaffirms the country’s leadership in adopting advanced pharmaceutical technologies. It strengthens international confidence in the UAE’s regulatory model, which strikes a careful balance between speed, scientific rigour, and transparency, ensuring that innovative treatments reach patients in a timely and reliable manner.”

She concluded by saying: "The Emirates Drug Establishment will continue to support medical innovation and adopt modern therapies that promote quality of life, in line with the nation's vision to build a state-of-the-art healthcare sector driven by knowledge, efficiency, and sustainability."

For his part, Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, stressed that the authorisation of Itvisma in the UAE is first and foremost about serving patients and their families.

He pointed out that close cooperation with the Emirates Drug Establishment and partners aims to ensure fast, equitable access to the treatment, providing people living with SMA who were previously unable to benefit from a one-time gene therapy a new opportunity to receive this type of medical intervention.

Ezz Eldin added that the UAE's support for accelerating the adoption of innovative treatments will contribute to changing the disease trajectory of spinal muscular atrophy, expressing the company's pride in its role in this medical advancement.

He noted that this achievement reflects Novartis’s commitment to neurology, building on its extensive experience in treating SMA and multiple sclerosis, while expanding its focus into neuroimmunology, neurodegenerative disorders, and neuromuscular diseases.

Itvisma’s approval is a strategic milestone within the Emirates Drug Establishment’s mission to position the UAE as a regional hub for pharmaceutical innovation by delivering advanced genetic therapies that meet the highest standards of quality and safety.

Adopting a modern regulatory model driven by precise governance, transparency, and international partnerships, the Establishment accelerates research, development, and the adoption of cutting-edge technologies and integrated care pathways.

This approach ensures timely patient access to innovative medicines, strengthens the nation’s healthcare readiness, and aligns with the UAE’s vision for a leading and sustainable healthcare system.