By VETTAPHARMA reporter – Derek Roche: The European Commission (EC) has approved Novartis’ Itvisma® (onasemnogene abeparvovec) for the treatment of children aged two years and older, adolescents, and adults with 5q spinal muscular atrophy (SMA) and a bi-allelic mutation in the SMN1 gene.
The approval makes Itvisma the first and only gene replacement therapy authorized in the European Union for this broad patient population and extends Novartis’ gene therapy portfolio across all age groups living with spinal muscular atrophy.
Itvisma is a one-time, fixed-dose intrathecal gene replacement therapy designed to address the genetic root cause of spinal muscular atrophy by delivering a functional copy of the SMN1 gene. Unlike therapies requiring ongoing administration, Itvisma provides a single-dose treatment that does not require adjustment for age or body weight and is intended to deliver sustained SMN protein expression to improve motor function.
The approval was supported by data from the registrational Phase 3 STEER study, together with the supportive Phase IIIb STRENGTH and Phase I/II STRONG studies. In the pivotal STEER trial, Itvisma demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE), with treatment effects sustained through 52 weeks of follow-up. The STEER and STRENGTH studies also demonstrated clinically meaningful improvements in both treatment-naïve and previously treated patients.
Spinal muscular atrophy is a rare inherited neuromuscular disorder caused by mutations or deletion of the SMN1 gene, resulting in progressive loss of motor neurons and muscle weakness affecting movement, swallowing, and breathing. According to Novartis, the disease has an estimated global prevalence of 1–2 per 100,000 people and an incidence of approximately 1 in 10,000 live births.
The most commonly reported adverse reactions with Itvisma include upper respiratory tract infection, pyrexia, vomiting, headache, and elevated hepatic enzymes. Novartis stated that, together with Zolgensma, Itvisma now enables gene replacement therapy options across the full spectrum of spinal muscular atrophy in Europe, from newborns to adults.
Quick FAQs
1. What Is Itvisma?
Itvisma® (onasemnogene abeparvovec) is a one-time intrathecal gene replacement therapy designed to treat spinal muscular atrophy by delivering a functional copy of the SMN1 gene.
2. What Happened?
The European Commission approved Novartis’ Itvisma for children aged two years and older, adolescents, and adults with 5q spinal muscular atrophy and bi-allelic SMN1 mutations.
3. What Studies Supported The Approval?
The approval was based on the registrational Phase 3 STEER study and supported by the Phase IIIb STRENGTH and Phase I/II STRONG studies, which evaluated the efficacy and safety of Itvisma in patients with spinal muscular atrophy.
4. What Is Spinal Muscular Atrophy?
Spinal muscular atrophy is a rare genetic neuromuscular disease caused by mutations or loss of the SMN1 gene, leading to progressive muscle weakness due to degeneration of motor neurons.
5. What Were The Key Results?
In the pivotal STEER study, Itvisma achieved a 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded, with benefits maintained through 52 weeks. Supportive studies also demonstrated clinically meaningful improvements in both treatment-naïve and previously treated patients.
6. Why Is This Important?
Itvisma becomes the first gene replacement therapy approved in the European Union for older children, adolescents, and adults with spinal muscular atrophy, expanding one-time treatment options across all age groups living with the disease.
