European regulators have recommended approval of Itvisma, a gene therapy for spinal muscular atrophy, marking another step forward in the development of advanced treatments for rare neuromuscular diseases.
The Committee for Medicinal Products for Human Use adopted a positive opinion on 23 April 2026, supporting the granting of a marketing authorisation for the therapy. As an advanced therapy medicinal product, the assessment was conducted in collaboration with the Committee for Advanced Therapies, reflecting the complexity of gene-based treatments.
Developed by Novartis Europharm Limited, Itvisma contains the active substance onasemnogene abeparvovec, a gene therapy designed to address the underlying genetic cause of spinal muscular atrophy. The treatment uses a non-replicating viral vector to deliver a functional copy of the survival motor neuron 1 gene into cells, enabling production of the SMN protein that is essential for motor neuron survival.
The therapy is intended for patients aged two years and older with 5q spinal muscular atrophy who have a bi-allelic mutation in the SMN1 gene. Clinical data indicate that treatment leads to improvements in motor function, offering a potential disease-modifying option for affected children.
Itvisma is administered as an injection and must be delivered in specialised clinical centres under the supervision of physicians experienced in managing spinal muscular atrophy. The most commonly reported side effects include upper respiratory tract infections, fever, vomiting, headache and elevated liver enzymes.
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The therapy had previously been designated as an orphan medicine during development. European authorities will now review whether this designation should be maintained following approval.
The positive opinion represents a key regulatory milestone, although final authorisation will depend on a decision by the European Commission, which typically follows within several weeks. Once granted, detailed prescribing information will be published to guide clinicians on its use in practice.
This news item has been summarised using AI and checked by humans before publication.
Sources
ema.europa.eu
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