EC approves Novartis’s Itvisma as first gene therapy for older SMA patients

Edited by Robert McTiernan··3 min read

The European Commission has approved Itvisma (onasemnogene abeparvovec) for the treatment of children aged two years and older, teenagers, and adults with 5q spinal muscular atrophy (SMA) carrying a bi-allelic mutation in the SMN1 gene — marking the first gene replacement therapy approved in the EU for this broader SMA population. The decision extends Novartis’s gene therapy franchise beyond infants, where onasemnogene abeparvovec was already established under the Zolgensma brand, into a substantially larger and more heterogeneous patient group that has historically been managed with chronic dosing therapies.

Itvisma is an intrathecal formulation of onasemnogene abeparvovec, whereas Zolgensma is administered intravenously to infants. Intrathecal delivery avoids the systemic vector exposure associated with intravenous gene therapy and enables treatment of larger, older patients for whom IV administration is not practical. Itvisma delivers a functional copy of the SMN1 gene via a single intrathecal injection using an adeno-associated virus 9 (AAV9) vector, designed to restore SMN protein expression in motor neurons. The fixed, weight-independent dose distinguishes it mechanistically and practically from approved alternatives: nusinersen (Spinraza), an antisense oligonucleotide requiring intrathecal administration every four months indefinitely, and risdiplam (Evrysdi), a daily oral SMN2 splicing modifier from Roche. The one-time administration model carries both clinical appeal and economic complexity, particularly in access negotiations across EU member states.

The EC approval rests primarily on data from the registrational STEER study, a Phase III trial in treatment-naive and pre-treated patients with later-onset SMA. In STEER, Itvisma demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE) score sustained over 52 weeks — a clinically meaningful gain in a population where motor function stabilization is often the realistic therapeutic ceiling. Supportive evidence came from the Phase IIIb STRENGTH study and the Phase I/II STRONG study, with results from STEER and STRENGTH published in Nature Medicine. The CHMP positive opinion that preceded this EC decision was issued at the June 2026 committee meeting.

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The competitive landscape in SMA has grown considerably more crowded. Biogen’s nusinersen recently received FDA approval for a high-dose regimen, reinforcing the continued clinical relevance of chronic ASO therapy in older patients. Biogen’s pipeline candidate salanersen has also received FDA Breakthrough Therapy Designation, targeting patients — including those with suboptimal outcomes following gene therapy — a population that may grow as Itvisma’s use expands. Not all pipeline efforts have succeeded: Roche halted development of emugrobart in SMA after lackluster findings, illustrating the difficulty of improving on existing approved standards.

For Novartis, extends Novartis’ gene therapy franchise across the SMA age spectrum in Europe — Zolgensma for newborns and infants, Itvisma for the older population — a positioning that no other company currently holds in the EU. Whether that breadth translates into clinical adoption will depend substantially on reimbursement outcomes and on how neurologists weigh a one-time intervention against the established track record of chronic therapies in adolescent and adult patients.

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