Global pharma giant Novartis has secured European Commission's approval for Itvisma (onasemnogene abeparvovec) -- the first & only gene replacement therapy for children, teens and adults living with spinal muscular atrophy (SMA).
The decision significantly expands access to one-time gene replacement therapy for a broader SMA population, offering a new treatment option for patients beyond early childhood.
Itvisma is designed to target the underlying genetic cause of SMA by replacing the faulty SMN1 gene through a one-time fixed dose that does not require adjustment for age or body weight. By restoring the missing gene, the therapy aims to improve motor function while offering an alternative to treatments that require ongoing dosing.
The approval is backed by results from the pivotal STEER trial, alongside supportive data from the Phase IIIb STRENGTH and Phase I/II STRONG studies.
In the registrational STEER study, Itvisma achieved a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale (HFMSE), with benefits sustained through 52 weeks of follow-up. Findings from both the STEER and STRENGTH studies also demonstrated clinically meaningful benefits in treatment-naïve and previously treated patients.
"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.”
“This approval marks a major milestone for people living with SMA,” said Patrick Horber, 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.”
