Overview
Novartis said the European Commission has approved Itvisma (onasemnogene abeparvovec) for use in patients with 5q spinal muscular atrophy who have a bi-allelic mutation in the survival motor neuron 1 gene. The approval extends to children aged two years and older, as well as teenagers and adults, making Itvisma the first gene replacement therapy authorized in the European Union for this broader SMA population.
Treatment profile
Itvisma is delivered as a one-time, fixed dose intended to address the underlying genetic cause of SMA by replacing the SMN1 gene. According to Novartis, this dosing approach does not require modification based on age or body weight and aims to improve motor function by restoring functional SMN1 gene expression.
Clinical evidence supporting approval
The European clearance is based primarily on results from the registrational STEER study, along with supportive data from the Phase IIIb STRENGTH trial and the Phase I/II STRONG study. In STEER, patients treated with Itvisma experienced a 2.39-point improvement on the Hammersmith Functional Motor Scale, with that benefit maintained over a 52-week observation period. Both the STEER and STRENGTH studies reportedly showed benefit in patients who were treatment-naive as well as those who had received prior therapy.
Reactions
Nicole Gusset, CEO of SMA Europe, described the approval as an important milestone for the SMA community, saying it brings the prospect of an additional treatment choice closer to people and families seeking options that reflect individual needs and circumstances. Patrick Horber, President of International at Novartis, said the authorization enables the company to provide gene replacement therapy options across different stages of SMA in Europe, from newborns to adults, when considered alongside their Zolgensma therapy.
Implications
This authorization positions Itvisma as a new therapeutic option in the EU for a wide age range of SMA patients with a defined genetic diagnosis. The decision rests on the specific clinical measures reported in the cited studies and the durability of benefit observed through one year in STEER.
Key points
- Itvisma (onasemnogene abeparvovec) approved by the European Commission for children aged two years and older, teenagers and adults with 5q SMA and a bi-allelic SMN1 mutation.
- The therapy is a one-time fixed dose gene replacement treatment that does not require age or weight-based dose adjustments.
- Approval was supported by data from the STEER registrational study and supportive STRENGTH and STRONG trials; STEER showed a 2.39-point improvement on the Hammersmith Functional Motor Scale sustained over 52 weeks.
Risks and uncertainties
- The primary efficacy readout cited was the 2.39-point improvement on the Hammersmith Functional Motor Scale with follow-up reported through 52 weeks; longer-term outcomes beyond this period were not detailed in the information provided.
- The approval and reported benefits are based on the STEER, STRENGTH and STRONG datasets; the scope of evidence reflected in those studies is the basis for the regulatory decision.