EU Approves Novartis’ Itvisma as First Gene Replacement Therapy for Children, Teens, and Adults with Spinal Muscular Atrophy

The European Commission has approved Itvisma® (onasemnogene abeparvovec) for the treatment of children aged two years and older, adolescents, and adults with 5q spinal muscular atrophy (SMA) carrying bi-allelic mutations in the SMN1 gene. The decision makes Itvisma the first gene replacement therapy authorized in the European Union for this broad patient population, extending gene therapy beyond infants and young children.

The approval also enables Novartis to offer gene replacement therapies across all age groups in Europe, with Zolgensma remaining available for younger patients and Itvisma expanding treatment options for older individuals living with SMA.

One-time gene replacement targets the genetic cause of SMA

SMA is a rare inherited neuromuscular disorder caused by mutations or deletion of the SMN1 gene, resulting in insufficient survival motor neuron (SMN) protein production. Progressive loss of motor neurons leads to worsening muscle weakness that affects movement, swallowing, and breathing.

Unlike therapies that require repeated administration, Itvisma delivers a functional copy of the SMN1 gene through a single intrathecal injection using an adeno-associated virus serotype 9 (AAV9) vector. The therapy uses a fixed dose that does not require adjustment according to age or body weight and aims to restore sustained SMN protein expression to preserve or improve motor function.

 Phase III program demonstrated durable motor function improvement

The approval is supported by findings from the registrational Phase III STEER trial, together with evidence from the Phase IIIb STRENGTH and Phase I/II STRONG studies.

In STEER (NCT05089656), treatment with Itvisma produced a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE) compared with control, with clinical benefits maintained through 52 weeks of follow-up.

The STRENGTH (NCT05386680) and STRONG (NCT03381729) studies further demonstrated clinically meaningful improvements in both treatment-naïve patients and those previously treated with other SMA therapies, supporting the therapy’s use across a wider spectrum of disease.

The overall safety profile remained consistent across studies. The most frequently reported adverse events included upper respiratory tract infection, pyrexia, vomiting, headache, and elevated hepatic enzymes.

Clinicians and patient groups welcome broader treatment choice

Nicole Gusset, Chief Executive Officer of SMA Europe, said the approval provides families with an additional therapeutic option that better reflects individual treatment preferences while underscoring the importance of timely and equitable access across European countries.

Professor Jana Haberlová, Head of the Neuromuscular Centre at Motol and Homolka University Hospital in Prague, noted that maintaining or improving motor function can substantially affect quality of life for older children, adolescents, and adults with SMA. She said the approval gives clinicians another therapeutic option to support patients throughout the disease course.

Patrick Horber, MD, President of International at Novartis, said the authorization addresses longstanding unmet needs by expanding access to one-time gene replacement therapy for older patients while complementing the company’s existing SMA treatment portfolio.

Commercial rollout to follow national reimbursement decisions

Following European Commission approval, Itvisma becomes eligible for commercialization across European Union member states. Patient access will now depend on country-specific pricing and reimbursement negotiations.

The approval further strengthens Novartis’ SMA franchise and broadens the role of gene replacement therapy across the continuum of care, offering eligible patients a one-time treatment option that directly addresses the underlying genetic cause of the disease.

What This Approval Means for Patients

The European Commission’s decision extends gene replacement therapy to children aged two and older, adolescents, and adults with Spinal Muscular Atrophy, offering a one‑time treatment that directly addresses the genetic cause of the disease. Unlike therapies requiring lifelong dosing, Itvisma® provides a single intrathecal administration that may preserve or improve motor function, though outcomes depend on disease stage. Access will vary across EU countries based on national reimbursement decisions.

Reference

Novartis receives European Commission approval for Itvisma® for spinal muscular atrophy (SMA) | Novartis