The European Commission has granted approval for Novartis’ Itvisma (onasemnogene abeparvovec) as a treatment for children two years and older, teenagers and adults who have 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene.
With this decision, Itvisma becomes the only gene replacement therapy which is currently approved for this wider patient population within the EU.
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Itvisma works by providing a functional copy of the SMN1 gene, which is deficient in people with SMA.
The therapy involves a one-off, fixed-dose intrathecal injection and does not require adjustments based on age or body weight.
The approval is supported by data from the registrational STEER trial, alongside results from the Phase IIIb STRENGTH and Phase I/II STRONG studies.
In the STEER study, Itvisma led to a statistically significant improvement of 2.39 points in the Hammersmith Functional Motor Scale, with this benefit maintained over a 52-week period.
Both the STEER and STRENGTH studies reported clinically meaningful benefits for patients, regardless of whether they were previously treated or treatment-naïve.
Novartis International president Patrick Horber said: “This approval marks a major milestone for people living with SMA. 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.”
Common side effects reported for Itvisma include upper respiratory tract infection, raised temperature, vomiting, headache and increased hepatic enzymes.
Novartis holds exclusive global licences related to the intravenous and intrathecal delivery of AAV9-based gene therapies for SMA.
In March 2026, Novartis received US Food and Drug Administration approval for Cosentyx (secukinumab) to treat paediatric patients aged 12 years and above with moderate to severe hidradenitis suppurativa.
