News: Abu Dhabi’s Sheikh Khalifa Medical City became the first hospital to administer ITVISMA after UAE regulatory approval in November 2025.
About ITVISMA Gene Therapy
- “Itvisma” is a brand name for the gene therapy drug onasemnogene abeparvovec-brve.
- It is a one-time gene therapy approved in late 2025 for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 years and older.
- The therapy addresses the genetic cause of the disease rather than only managing symptoms.
- Developed by: ITVISMA was developed by Novartis.
- Aim: The treatment aims to improve motor function and reduce long-term treatment dependence.
- Mechanism
- SMN1 Gene: This is the gene that is mutated or missing in people with spinal muscular atrophy (SMA), the condition Itvisma treats.
- The gene’s role is to produce the essential SMN protein needed for motor neuron function, muscle movement, breathing, and swallowing.
- ITVISMA’s role: It is an adeno-associated virus (AAV) vector-based therapy designed to deliver a new, working copy of the SMN1 gene directly to the motor neurons in the spinal cord via a one-time intrathecal injection.
- Result: The new gene enables continuous and sustained production of the SMN protein, addressing the genetic root cause of SMA to improve or stabilize motor function.
- Risk of Therapy
- Common Side Effects: Common side effects include fever, vomiting, headache, and upper respiratory tract infections.
- Serious Risks: Serious risks include liver toxicity, heart-related effects, and reduced platelet counts.
- Significance : ITVISMA can deliver sustained motor-function improvement with a single dose, reduce lifelong treatment dependence, and expand access beyond infant-only therapies.
