Novartis secures European Commission approval for Itvisma in spinal muscular atrophy

Novartis has recently announced that the European Commission has approved Itvisma (onasemnogene abeparvovec) for the treatment of children aged two and older, teenagers, and adults living with 5q spinal muscular atrophy carrying a bi-allelic mutation in the survival motor neuron 1 gene. With this decision, Itvisma becomes the first and only gene replacement therapy currently holding approval for this broad SMA population anywhere in the European Union.

Itvisma has been engineered to tackle the genetic origin of SMA through a single fixed dose that requires no adjustment for age or body weight. By delivering a functional copy of the SMN1 gene, it offers the potential to improve motor function through a one-time intervention that stands apart from the ongoing dosing schedules associated with other available therapies in this population.

“European approval is an important milestone for the SMA community. Beyond the scientific achievement, it brings the prospect of a new treatment option closer to people and families who are looking for choices that reflect their individual needs and circumstances. We welcome today’s decision and hope it translates into timely and equitable access across Europe,” said Nicole Gusset, CEO of SMA Europe.

“Maintaining or improving motor function can make a meaningful difference for older children, teens and adults living with SMA,” said Professor Jana Haberlová, Head of Neuromuscular Centre at the Department of Paediatric Neurology, Motol and Homolka University Hospital, Prague, Czech Republic. “The approval of Itvisma in Europe is an important advance because it brings a new gene replacement therapy option to a broader patient population and gives clinicians an additional way to support patients across the course of the disease.”

The approval is grounded in data from the registrational STEER study, supported by findings from the Phase IIIb STRENGTH and Phase I/II STRONG studies. Within STEER, Itvisma achieved a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale, with benefits sustained across 52 weeks of follow-up. Both the STEER and STRENGTH studies showed clinically meaningful gains in treatment-naïve and previously treated patients alike.

“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.”

---

---

Related Topics and Keywords

itvisma, Novartis, spinal muscular atrophy