Itvisma®

Novartis receives European Commission approval for Itvisma® (onasemnogene abeparvovec) for the treatment of children two years of age and older, teenagers, and adults living with 5q spinal muscular atrophy (SMA) who have a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene. Announced from Basel on July 2, 2026, this landmark marketing authorization establishes the therapeutic option as the first and only gene replacement therapy currently approved for this broad patient demographic within the European Union. Uniquely designed to address the genetic root cause of SMA, the advanced therapy provides a functional copy to replace the faulty human SMN1 gene. Because it targets the root cause of the disease with a fixed, one-time dose that does not need to be adjusted for age or body weight, it offers a distinct paradigm shift compared to continuous, ongoing chronic dosing approaches. Spinal muscular atrophy is a rare, genetic neuromuscular disease caused by mutations or a complete deletion of the human SMN1 gene, which prevents the body from producing sufficient levels of functional survival motor neuron (SMN) protein. This specific protein is critically responsible for producing most of the SMN protein the body needs for proper muscle function, including breathing, swallowing, and basic motor movement. Without it, motor neurons undergo irreversible loss, resulting in progressive and debilitating muscle weakness across the body. A secondary gene, known as the SMN2 gene, produces only a small fraction, approximately 10%, of functional SMN protein compared to the healthy SMN1 gene, meaning that individuals who happen to carry more copies of the SMN2 gene generally manifest a less severe clinical form of the disease. Globally, SMA carries an estimated prevalence of roughly 1 to 2 individuals per 100,000 people, with an incidence rate occurring in approximately 1 out of every 10,000 live births. Patient advocacy groups and neuromuscular clinicians across the European continent have expressed profound enthusiasm regarding the therapeutic expansion enabled by this regulatory milestone. Nicole Gusset, Chief Executive Officer of SMA Europe, highlighted that the European approval is an important milestone for the SMA community because, beyond the scientific achievement, it brings the prospect of a new treatment option closer to people and families who are looking for choices reflecting individual needs and circumstances. Professor Jana Haberlová, Head of the Neuromuscular Centre at the Department of Paediatric Neurology, Motol and Homolka University Hospital in Prague, Czech Republic, emphasized that maintaining or improving motor function can make a meaningful difference for older children, teens, and adults living with SMA. She explained that the approval 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. Clinical Study Data Demonstrating Why Novartis receives European Commission approval for Itvisma The formal regulatory decision is fundamentally backed by clinical evidence from the registrational Phase III STEER trial, along with vital supportive evidence gathered from the Phase IIIb STRENGTH trial and the Phase I/II STRONG trial. The clinical trial program heavily emphasizes the efficacy achieved before Novartis receives European Commission approval for Itvisma. In the primary STEER study, which evaluated treatment-naïve patients, the gene therapy demonstrated a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale (HFMSE) compared against the study's control group, with therapeutic benefits shown to be fully sustained throughout a comprehensive 52-week post-treatment follow-up observation window. Furthermore, data collected across both the STEER and STRENGTH clinical protocols confirmed a clinically meaningful benefit for both treatment-naïve individuals and pre-treated patients who had previously undergone alternative chronic therapy regimens. With the confirmation that Novartis receives European Commission approval for Itvisma, eligible patients have a one-time treatment alternative to continuous chronic dosing therapies. Patrick Horber, MD, President, International at Novartis, added that this approval marks a major milestone for people living with SMA because it expands access to a one-time gene replacement therapy for older children, teens, and adults—potentially addressing long-standing unmet needs for patients. He noted that, together with Zolgensma, Novartis can now offer gene replacement therapy options across different stages of SMA in Europe, extending from newborns to adults. For clinical guidance and details on therapeutic protocols within our network, healthcare practitioners can review the internal SMA Therapeutic Framework database. This therapeutic addition leverages advanced molecular technologies under exclusive licensing agreements. Novartis maintains an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene replacement therapy for the treatment of all types of SMA. Additionally, the company holds an exclusive worldwide license from REGENXBIO for any recombinant adeno-associated virus (AAV) vector within its intellectual property portfolio. Full prescribing guidelines and the summary of product characteristics will be continuously maintained and updated via the official link to the European Medicines Agency portal. Clinical Trial Identification and Motor Function Metrics Clinical Trial Registry Identifier / TypeStudy NamePatient Target FocusPrimary Motor Function Metric & FindingsRegistrational Phase IIISTEER TrialTreatment-naïve patientsStatistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale (HFMSE) with clinical effects fully sustained over a 52-week post-treatment follow-up window.Phase IIIb SupportiveSTRENGTH TrialPre-treated and treatment-naïve patientsDemonstrated clinically meaningful therapeutic benefits, supporting application across a wider disease spectrum.Phase I/II SupportiveSTRONG TrialDiverse patient cohortsDemonstrated clinically meaningful therapeutic benefits and motor improvements across studied cohorts. Product Properties and Disease Demographics Regulatory / Clinical ParameterSourced Fact / Value Specification from NovartisDeveloper & ManufacturerNovartisActive Substance Generic Nameonasemnogene abeparvovecApproved Indication ProfileChildren 2 years and older, teens, and adults living with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 geneDelivery Vector & Dose TypeIntrathecal delivery of AAV9 gene replacement therapy; fixed, one-time doseDose Adjustment RequirementZero adjustment required for age or body weightGlobal Disease PrevalenceApproximately 1 to 2 individuals per 100,000 peopleGlobal Disease IncidenceRoughly 1 out of every 10,000 live birthsSMN2 Gene Functional CapacityProduces a small fraction (~10%) of functional SMN protein compared with the SMN1 geneLicensing Framework PartnersNationwide Children's Hospital (exclusive worldwide license for intravenous/intrathecal AAV9 delivery); REGENXBIO (exclusive worldwide license for recombinant AAV vectors)

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Novartis Receives European Commission Approval for Itvisma for Spinal Muscular Atrophy

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Novartis Receives European Commission Approval for Itvisma for Spinal Muscular Atrophy

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