Novartis wins EC approval for Itvisma in broader SMA population
Novartis has secured European Commission approval for Itvisma (onasemnogene abeparvovec), making it the first gene replacement therapy authorised in the EU for a broad spinal muscular atrophy (SMA) population spanning children aged two and older through to adults. The decision, announced on 30 June 2026, substantially extends the reach of gene-based SMA treatment beyond the newborn and infant populations previously served by existing approvals.
Itvisma is an adeno-associated virus 9 (AAV9)-based therapy that delivers a functional copy of the faulty SMN1 gene via a single intrathecal injection, with no dose adjustment required for age or body weight. Novartis positions this fixed-dose approach as a structural advantage over other SMA therapies that require ongoing administration.
Clinical evidence
The EC approval is underpinned primarily by the STEER registrational study, with supportive data from the Phase IIIb STRENGTH trial and the Phase I/II STRONG study. In STEER, Itvisma produced a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale (HFMSE) sustained over 52 weeks. Both STEER and STRENGTH demonstrated clinically meaningful benefit in treatment-naïve and previously treated patients, with results from both studies published in Nature Medicine in 2025.
Professor Jana Haberlová, Head of the Neuromuscular Centre at Motol and Homolka University Hospital in Prague, said: "The approval of Itvisma in Europe is an important advance because it brings a new gene replacement therapy option to a broader patient population and gives clinicians an additional way to support patients across the course of the disease."
The most commonly reported adverse events in clinical studies included upper respiratory tract infection, pyrexia, vomiting, headache, and elevated hepatic enzymes, consistent with the profile seen in AAV9-based gene therapies more broadly.
Market context and competitive landscape
With Itvisma now approved, Novartis can offer gene replacement therapy options across the full SMA age spectrum in Europe. Its earlier product Zolgensma, also an AAV9-based SMN1 gene therapy but delivered intravenously and approved for patients under 21 kilograms, covers the neonatal and infant segment. The two products together give Novartis a distinctive position in the European SMA market.
SMA treatment has evolved rapidly since the approval of the splice-modifying therapy nusinersen (Biogen's Spinraza) and the small-molecule risdiplam (Roche's Evrysdi), both of which require chronic dosing. The appeal of a one-time therapy is clear for patients and families, though access will depend on pricing negotiations with individual EU member state reimbursement bodies, which often proceed more slowly than EC marketing authorisation itself. Payers will scrutinise long-term durability data closely, given that the sustained SMN protein expression assumed from a single dose has not yet been tracked beyond several years in the older population.
SMA affects an estimated one to two people per 100,000 globally, with an incidence of roughly one in 10,000 live births, making reimbursement negotiations particularly sensitive to cost-per-patient arguments in rare disease frameworks. Novartis holds exclusive worldwide licences for AAV9-based SMA gene replacement therapy from Nationwide Children's Hospital, REGENXBIO, and Généthon, providing broad intellectual property protection around both intravenous and intrathecal delivery routes.
Near-term attention will focus on the pace of national reimbursement decisions across major EU markets and on further long-term follow-up data from the STEER and STRENGTH cohorts to inform real-world treatment sequencing decisions for patients already on chronic SMA therapies.