European Commission Approves Itvisma Gene Therapy for Older Children, Teens and Adults with SMA

The European Commission has approved Itvisma (onasemnogene abeparvovec, given by spinal injection) for children aged two and over, teenagers and adults living with 5q spinal muscular atrophy who have a bi-allelic mutation in the SMN1 gene. The decision, confirmed on 2 July 2026, follows a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in April 2026.

It's the first time a one-off gene replacement therapy has been approved anywhere in the EU for this broader group of older children, teenagers and adults with SMA — until now, gene therapy in Europe has only been available to infants under two, dosed intravenously as Zolgensma.

How is this different from Zolgensma?

Itvisma uses the same active substance as Zolgensma (onasemnogene abeparvovec) but is delivered directly into the spinal canal (intrathecally) rather than into a vein. Because the dose doesn't need to scale with body weight in the same way, this route makes it possible to treat older, larger children and adults safely — something the intravenous form isn't suitable for.

Novartis says the EU approval is supported by data from its Phase III STEER trial in treatment-naive and previously treated patients with later-onset SMA, in which patients on Itvisma showed a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE), sustained over 52 weeks. Supportive data also came from the Phase 3b STRENGTH study (in patients switching from nusinersen or risdiplam) and the earlier Phase I/II STRONG study.

What this means for the UK

This is an EU regulatory decision, so it doesn't itself change anything for families in the UK — Itvisma is not yet licensed here by the MHRA. However, it's a significant step in a process TreatSMA has been following closely: NICE opened its own evaluation of intrathecal gene therapy for SMA back in April 2026, and this EU approval, alongside the therapy's FDA approval in the US in November 2025, adds substantially to the international evidence and real-world backdrop that any future UK licensing and NICE decision would draw on.

We'll continue to track progress through the MHRA and NICE and report back as soon as there's UK-specific news.

Sources

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