EU Approves Novartis’ Itvisma for Broad SMA Patient Population

Novartis has received European Commission approval for Itvisma® (onasemnogene abeparvovec), expanding access to gene replacement therapy for children, teenagers and adults living with 5q spinal muscular atrophy (SMA).

The approval covers patients aged two years and older with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene, the genetic cause of the most common form of SMA. According to Novartis, Itvisma is now the first and only gene replacement therapy approved in the European Union for this broader SMA population.

SMA is a rare inherited neuromuscular disease that causes progressive muscle weakness and loss of motor function. While treatment options have improved significantly in recent years, many therapies require ongoing administration. Itvisma is designed as a one-time fixed-dose treatment, meaning the dose does not need to be adjusted according to a patient’s age or body weight.

The therapy works by delivering a functional copy of the SMN1 gene, with the aim of addressing the underlying genetic cause of the disease. Novartis said the treatment could provide an alternative for patients and families seeking a one-time treatment option rather than long-term dosing schedules.

The European Commission’s decision was supported by data from the registrational Phase III STEER study, alongside findings from the Phase IIIb STRENGTH study and the Phase I/II STRONG study. In the STEER trial, patients treated with Itvisma showed a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded, or HFMSE, compared with the study control group. The benefit was sustained through 52 weeks of follow-up.

The STEER and STRENGTH studies also indicated clinically meaningful improvements in motor function among both treatment-naïve patients and those who had previously received other SMA therapies.

Nicole Gusset, chief executive of SMA Europe, said the approval offers new hope for people living with SMA and their families. She added that timely and equitable access across European countries will be essential for the therapy’s impact.

Professor Jana Haberlová of Motol and Homolka University Hospital in Prague said maintaining or improving motor function can have a meaningful effect on quality of life for older children, teenagers and adults with SMA.

Novartis said Itvisma complements its existing SMA gene therapy, Zolgensma, potentially allowing the company to offer gene replacement treatment options across a wider range of SMA patients in Europe.