Novartis has received European Commission approval for Itvisma (onasemnogene abeparvovec) to treat patients aged two years and older with 5q spinal muscular atrophy (SMA) carrying bi-allelic mutations in the SMN1 gene.
The decision expands access to gene replacement therapy across a broader SMA population in the European Union, including older children, adolescents, and adults. Itvisma is the first gene replacement therapy approved in the EU for this wider age group, complementing existing options that primarily target younger patients.
Itvisma is designed as a single-dose therapy intended to address the underlying genetic cause of SMA by delivering a functional copy of the SMN1 gene. Unlike chronic treatment regimens, the therapy is administered once and does not require dose adjustments based on age or body weight.
The approval is supported by data from the Phase III STEER trial, along with results from the Phase IIIb STRENGTH and Phase I/II STRONG studies. In STEER, patients treated with Itvisma showed a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded over 52 weeks. Additional data indicated clinically meaningful benefit in both treatment-naïve and previously treated patients.
SMA is a rare neuromuscular disorder characterized by progressive muscle weakness due to degeneration of motor neurons. It affects approximately 1 to 2 per 100,000 people globally.
With this authorization, Novartis now has gene therapy options for SMA spanning multiple age groups in Europe.
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