Basel, Switzerland, July 2, 2026
Novartis has achieved a major milestone in the treatment of spinal muscular atrophy (SMA) after receiving European Commission (EC) approval for Itvisma® (onasemnogene abeparvovec), making it the first and only gene replacement therapy approved in the European Union for children aged two years and older, teenagers, and adults living with 5q SMA caused by a bi-allelic mutation in the SMN1 gene. The approval significantly expands access to one-time gene replacement therapy, providing patients across a broader age range with an innovative treatment designed to address the genetic root cause of SMA rather than managing symptoms through continuous dosing. With this latest authorization, Novartis now offers gene replacement therapy options across every age group affected by SMA in Europe, strengthening its leadership in rare neurological diseases and advancing personalized treatment choices for patients and healthcare providers. The approval is supported by robust clinical evidence demonstrating meaningful improvements in motor function and long-term therapeutic benefit.
One-Time Gene Therapy Expands Treatment Options for SMA Patients
Itvisma® is an AAV9-based gene replacement therapy developed to deliver a functional copy of the defective SMN1 gene, enabling sustained production of survival motor neuron (SMN) protein through a single intrathecal administration. Unlike therapies requiring repeated lifelong dosing, Itvisma is administered as a fixed one-time dose that does not require adjustment based on age or body weight, offering a differentiated treatment option for eligible patients. SMA is a rare, progressive neuromuscular disorder that causes irreversible motor neuron loss, leading to worsening muscle weakness affecting movement, swallowing, and breathing.
By directly replacing the faulty gene responsible for the disease, Itvisma aims to improve motor function while addressing the underlying biological cause of SMA. Patient advocacy organizations and clinical experts welcomed the approval, highlighting its importance in expanding therapeutic choice and providing physicians with an additional option for managing older children, adolescents, and adults living with this debilitating genetic condition.
Clinical Studies Demonstrate Significant Functional Improvement
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The European Commission approval is based on positive findings from the registrational Phase III STEER study, supported by data from the Phase IIIb STRENGTH and Phase I/II STRONG clinical trials. In the pivotal STEER study, patients treated with Itvisma achieved a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE), with benefits maintained throughout 52 weeks of follow-up. Both treatment-naïve individuals and patients previously treated with other SMA therapies demonstrated clinically meaningful improvements in motor function, supporting the therapy’s effectiveness across a broad patient population.
The clinical program also confirmed a manageable safety profile, with the most commonly reported adverse events including upper respiratory tract infection, fever, vomiting, headache, and elevated liver enzymes. These encouraging outcomes reinforce the potential of gene replacement therapy to improve long-term functional outcomes while reducing disease progression in patients living with SMA.
Novartis Strengthens Leadership in Gene Replacement Therapy
The approval further expands Novartis’ neuroscience and gene therapy portfolio, complementing Zolgensma® and enabling the company to provide gene replacement therapies across the full spectrum of SMA patients, from newborns through adulthood. Novartis continues to invest heavily in innovative treatments targeting neurological diseases, including multiple sclerosis, neuroimmunology, neurodegeneration, and neuromuscular disorders, with the goal of improving long-term patient outcomes through transformative therapies.
By combining advanced gene therapy technology with growing clinical evidence, Itvisma represents another important step toward expanding access to precision medicine for rare genetic diseases. As European countries begin reimbursement and access discussions, the approval is expected to provide new hope for families affected by SMA while reinforcing Novartis’ commitment to delivering innovative, one-time therapies capable of addressing serious inherited disorders at their genetic source.
Source: Novartis press release
