Novartis announced that the European Commission (EC) has approved Itvisma® (onasemnogene abeparvovec) for treating children aged two years and older, teens and adults with 5q spinal muscular atrophy (SMA) who have a bi-allelic mutation in the SMN1 gene.

The approval makes Itvisma the first and only gene replacement therapy approved in the European Union for this broad SMA patient population.

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Itvisma is a one-time, fixed-dose gene replacement therapy designed to replace the faulty SMN1 gene, targeting the genetic cause of SMA without requiring dose adjustments based on age or body weight.

The approval is supported by data from the STEER, STRENGTH, and STRONG clinical studies. In the pivotal STEER trial, Itvisma demonstrated a statistically significant 2.39-point improvement in motor function, with benefits maintained over 52 weeks. The studies also showed clinically meaningful improvements in both treatment-naïve and previously treated patients.

“This approval marks a major milestone for people living with SMA,” said Patrick Horber, MD, President, International, Novartis. “With Itvisma, we are going further to expand access to a one-time gene replacement therapy for older children, teens and adults – potentially addressing long-standing unmet needs for patients. Together with Zolgensma, we can now offer gene replacement therapy options across different stages of SMA in Europe, from newborns to adults.”

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