European Commission Expands Approval of Novartis' Itvisma for Broader SMA Patient Population

The decision makes Itvisma the first and only gene replacement therapy approved in the EU for older children, teenagers and adults living with 5q spinal muscular atrophy.

The European Commission has approved Novartis' Itvisma (onasemnogene abeparvovec) for the treatment of older children, teenagers and adults with 5q spinal muscular atrophy (SMA) who have a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene.

The approval significantly expands access to gene replacement therapy within Europe, making Itvisma the only approved treatment of its kind for this broader patient population.

Expanding Access Beyond Early Childhood

Spinal muscular atrophy is a rare genetic neuromuscular disorder caused by mutations in the SMN1 gene, resulting in insufficient production of survival motor neuron protein and progressive muscle weakness.

Until now, gene replacement therapies have largely been focused on younger patients.

The European Commission's decision extends access to older children, adolescents and adults, addressing a long-standing unmet need for patients who previously had limited treatment options.

Itvisma delivers a functional copy of the SMN1 gene through a single fixed-dose intrathecal injection, eliminating the need for ongoing dosing adjustments based on age or body weight.

Clinical Evidence Supports Approval

The approval is supported by data from three clinical studies:

• STEER

• Phase IIIb STRENGTH

• Phase I/II STRONG
  

The registrational STEER study demonstrated a statistically significant improvement of 2.39 points on the Hammersmith Functional Motor Scale, with benefits maintained throughout a 52-week follow-up period.

Both the STEER and STRENGTH studies also showed clinically meaningful improvements in motor function across both treatment-naïve patients and those who had previously received SMA therapies.

These findings helped demonstrate that gene replacement therapy may provide meaningful functional benefits beyond the early stages of the disease.

A Milestone for Gene Therapy in SMA

Novartis believes the expanded indication represents an important step forward in broadening access to one-time gene replacement therapy.

The company said the approval has the potential to address significant unmet needs among older SMA patients who have historically had fewer therapeutic options than younger children.

As with any gene therapy, treatment is associated with potential side effects.

The most commonly reported adverse events include upper respiratory tract infection, fever, vomiting, headache and elevated liver enzymes.

Strengthening Novartis' Rare Disease Portfolio

The approval further reinforces Novartis' position in rare genetic diseases and gene therapy.

The company holds exclusive global licences covering both intravenous and intrathecal delivery of AAV9-based gene therapies for spinal muscular atrophy, positioning it as one of the leading developers in this space.

Gene therapies continue to reshape the treatment landscape for inherited neurological disorders by offering the potential for long-lasting therapeutic benefit following a single administration.

Summary

The European Commission has approved Novartis' Itvisma for older children, teenagers and adults with 5q spinal muscular atrophy, making it the first gene replacement therapy authorised for this wider patient population within the EU.

Supported by positive clinical data across multiple studies, the approval expands treatment options for patients with SMA while reinforcing the growing role of one-time gene therapies in managing rare neurological diseases.

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