Breaking News: Novartis receives European Commission market authorisation for ITVISMA
The European Commission has granted marketing authorisation for Itvisma (intrathecal formulation of onasemnogene abeparvovec, developed by Novartis) for the treatment of adults and children aged 2 years and older living with 5q spinal muscular atrophy (SMA).
The European Commission has granted marketing authorisation for Itvisma (intrathecal formulation of onasemnogene abeparvovec, developed by Novartis) for the treatment of adults and children aged 2 years and older living with 5q spinal muscular atrophy (SMA). The European Commission’s decision follows the positive opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) in April 2026.
Unlike Zolgensma, which is administered intravenously, Itvisma is delivered through a single intrathecal injection into the cerebrospinal fluid surrounding the spinal cord. Intrathecal route enables the therapy to be delivered directly to the central nervous system while a lower vector dose and without requiring weight-based dosing. This approach has enabled the therapy to be studied in older children, adolescents, and adults, leading to its approval for this broader age group.
For the SMA community, the European Commission’s decision represents a milestone in the regulatory process, following the positive CHMP opinion adopted in April 2026. The next steps will be national pricing, reimbursement, and access decisions, which determine when and how people can receive the treatment in each country.
For more information, click below.