Novartis has secured European Commission approval for Itvisma for the treatment of children aged two years and older, as well as teens and adults living with 5q spinal muscular atrophy with a bi-allelic mutation in the SMN1 gene.
The decision makes Itvisma the first gene replacement therapy authorised in the EU for this broad patient group.
The therapy is designed to address the genetic root cause of SMA through a fixed, one-time dose that does not require adjustment for age or body weight. By replacing the SMN1 gene, Itvisma aims to improve motor function and offers an alternative to ongoing dosing approaches used with other treatments.
Nicole Gusset, CEO of SMA Europe, said: “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.”
She added: “We welcome today’s decision and hope it translates into timely and equitable access across Europe.”
Professor Jana Haberlová, Head of Neuromuscular Centre at the Department of Paediatric Neurology, Motol and Homolka University Hospital, Prague, Czech Republic, said: “Maintaining or improving motor function can make a meaningful difference for older children, teens and adults living with SMA.”
She explained: “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 EC decision is supported by data from the registrational STEER study and the supportive phase 3b STRENGTH and phase 1/2 STRONG studies. In STEER, Itvisma delivered a statistically significant 2.39‑point improvement in HFMSE scores, with effects sustained over 52 weeks. Across STEER and STRENGTH, clinically meaningful benefit was observed in both treatment‑naïve and pre‑treated patients.
Patrick Horber, MD, President, International, Novartis, said: “This approval marks a major milestone for people living with SMA.”
He added: “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.”
