Abu Dhabi has achieved a historic milestone in global healthcare. For the first time in the world, ITVISMA gene therapy has been administered to a patient suffering from spinal muscular atrophy (SMA). The treatment marks a major leap in precision medicine and genomics-based healthcare.
What Is the News About?
- On December 28, 2025, Sheikh Khalifa Medical City successfully administered ITVISMA, a one-time gene therapy for SMA.
- The therapy was delivered under the supervision of the Department of Health, Abu Dhabi, following regulatory approval granted on November 25, 2025.
- With this step, the UAE became the second country after the United States to approve ITVISMA for clinical use.
About ITVISMA Gene Therapy
- ITVISMA is a next-generation gene therapy developed by Novartis.
- It directly targets the genetic cause of spinal muscular atrophy.
Key Features
- One time single dose gene therapy
- Replaces the defective SMN1 gene
- Enables production of Survival Motor Neuron (SMN) protein
- Approved for patients aged 2 years and above, including adults
- Expands access beyond infant-only SMA treatments
Understanding Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy is a rare genetic neuromuscular disorder.
It leads to,
- Progressive muscle weakness
- Loss of motor function
- Difficulty in breathing and swallowing
- Life-threatening complications in severe cases
SMA is caused by mutations in the SMN1 gene, which is essential for motor neuron survival.
Policy and Global Health Significance
- Positions Abu Dhabi as a leader in genomics and precision medicine
- Strengthens the UAE’s role in advanced rare disease care
- Demonstrates rapid regulatory and clinical capability
According to Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health, Abu Dhabi, the achievement reflects the emirate’s commitment to world-class healthcare delivery.
Cost and Accessibility Concerns
- The exact cost of ITVISMA has not been officially disclosed.
- However, it is expected to be comparable to Zolgensma, another SMA gene therapy priced at around $2 million per dose.
This highlights,
- Ongoing debates around affordability of rare disease therapies
- Need for innovative health financing models
- Importance of insurance and government support systems
Static Background: Gene Therapy in Medicine
Gene therapy involves inserting, altering, or replacing genes to treat disease.
It is increasingly used in,
- Rare genetic disorders
- Certain cancers
- Neuromuscular conditions
Gene therapies are considered a frontier area of modern medicine due to their potential for curative outcomes with a single intervention.
Key Data at a Glance
- Therapy Name: ITVISMA
- Disease targeted: Spinal Muscular Atrophy
- Developer: Novartis
- First administration: December 28, 2025
- First hospital globally: Sheikh Khalifa Medical City, Abu Dhabi
- UAE approval date: November 25, 2025
- Patient eligibility: 2 years and above
Question
Q. ITVISMA gene therapy targets which genetic defect?
A. DMD gene
B. BRCA gene
C. SMN1 gene
D. CFTR gene
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