Key Highlights
- The European Commission has approved Novartis’s Itvisma (onasemnogene abeparvovec) as the first and only gene replacement therapy in the EU for children two years and older, teens, and adults with 5q spinal muscular atrophy carrying a bi-allelic SMN1 mutation.
- Itvisma is delivered as a single, fixed one-time dose that does not require adjustment for age or body weight, offering a distinct alternative to therapies that require ongoing dosing for this broader SMA population.
- Approval rests on the registrational STEER study, which showed a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale sustained through 52 weeks, with Novartis now positioned to offer gene replacement options across the full age spectrum of SMA, from newborns through adulthood, alongside its existing therapy Zolgensma.
Closing a Long-Standing Treatment Gap
Itvisma’s approval extends gene replacement therapy to an SMA population that previously lacked access to this treatment approach, specifically older children, teens, and adults with the bi-allelic SMN1 mutation. By targeting the underlying genetic cause of the disease rather than managing symptoms through repeated dosing, the therapy offers clinicians a fundamentally different option for patients further along in the course of the disease.
A One-Time Dose Built for Simplicity
Unlike therapies that require ongoing administration, Itvisma is designed as a single intrathecal injection using a fixed dose that does not need to be recalibrated for a patient’s age or weight. This design aims to simplify treatment logistics for both patients and healthcare providers while delivering a functional copy of the SMN1 gene intended to support sustained motor neuron protein production.
Clinical Evidence Spanning Multiple Studies
The approval draws on data from the STEER registrational trial, supported by the Phase IIIb STRENGTH and Phase I/II STRONG studies. Beyond the sustained functional motor improvement observed in STEER, both STEER and STRENGTH demonstrated clinically meaningful benefit across treatment-naïve and previously treated patients, with the most common side effects including upper respiratory infection, fever, vomiting, headache, and elevated liver enzymes.
Expanding Novartis’s Neuroscience Commitment
Company and patient-advocacy leaders described the approval as a significant milestone for the broader SMA community, particularly for families seeking treatment options suited to individual needs at different disease stages. With Itvisma now cleared alongside Zolgensma, Novartis can offer gene replacement therapy across the full range of SMA patients in Europe, reinforcing the company’s stated focus on expanding innovation in neuromuscular disease and neurology more broadly.
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