Novartis has announced that the European Commission (EC) has approved Itvisma (onasemnogene abeparvovec) for the treatment of children aged 2 years and older, teens and adults living with 5q Spinal Muscular Atrophy (SMA) with a bi-allelic mutation in the Survival Motor Neuron 1 (SMN1) gene.

With this approval, Itvisma becomes the first and only gene replacement therapy currently approved for this broad SMA population in the European Union (EU).

Itvisma is uniquely designed to address the genetic root cause of SMA with a 1-time fixed dose that does not need to be adjusted for age or body weight. By replacing the SMN1 gene, Itvisma can improve motor function, offering a one-time treatment option distinct from ongoing dosing approaches associated with other available therapies for this population.

Nicole Gusset, CEO, 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. We welcome today’s decision and hope it translates into timely and equitable access across Europe.”

The approval is based on data from the registrational STEER study, and supportive phase 3b STRENGTH and phase 1/2 STRONG studies. In STEER, Itvisma demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale (HFMSE) with effects sustained over 52 weeks of follow-up. The STEER and STRENGTH studies also showed clinically meaningful benefit for treatment-naïve and pre-treated patients.

 Patrick Horber, MD, President, International, Novartis, said, “This approval marks a major milestone for people living with SMA. 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.”