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Gene therapy Itvisma approved in the EU for patients of all ages

Editorial illustration of a neurologist speaking with a young adult using a powered wheelchair, with a DNA strand and a map of Europe symbolizing the European Union approval of Itvisma gene therapy for spinal muscular atrophy (SMA).

The one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) has won expanded approval in the European Union for people with spinal muscular atrophy (SMA) aged 2 and older. The decision, valid across all EU member states, makes Itvisma the first and only gene therapy approved for this broad population and follows a positive recommendation from a committee of the European Medicines Agency (EMA). The therapy is developed by Novartis.

Unlike the older therapy Zolgensma (onasemnogene abeparvovec-xioi), which is given intravenously and is approved in Europe only for infants and young children (up to 21 kg), Itvisma is administered intrathecally — a direct injection into the spinal canal at a fixed dose. The approval is based mainly on data from the Phase 3 STEER study and the STRENGTH study; results showed significant improvement in, or stable maintenance of, motor function.

Source: SMA News Today

Gene therapy Itvisma approved in the EU for patients of all ages | SMA Moldova | SMA Moldova