Novartis Receives Positive CHMP Opinion for Itvisma Gene Therapy for Spinal Muscular Atrophy
Key Insights
The European Medicines Agency (search)'s Committee for Medicinal Products for Human Use has adopted a positive opinion recommending marketing authorization for Itvisma (search), Novartis' gene replacement therapy for spinal muscular atrophy (search) patients aged two years and older.
In the Phase III STEER study, Itvisma (search) demonstrated a statistically significant 2.39-point improvement in motor function on the Hammersmith Functional Motor Scale compared to 0.51 points for placebo (p=0.0074).
If approved, Itvisma (search) would become the first and only gene replacement therapy for children two years and older, teens, and adults with SMA (search) in the European Union, offering a one-time treatment option to replace the defective SMN1 (search) gene.
Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (search) (EMA) has adopted a positive opinion recommending marketing authorization for Itvisma (search)® (intrathecal onasemnogene abeparvovec) for the treatment of children two years and older, teens, and adults living with 5q spinal muscular atrophy (search) (SMA (search)) with a bi-allelic mutation in the survival motor neuron 1 (search) (SMN1 (search)) gene.
If approved by the European Commission, Itvisma (search) would become the first and only gene replacement therapy for this patient population in the European Union. The therapy is uniquely designed to address the genetic root cause of SMA (search) with a one-time fixed dose that does not require adjustment for age or body weight.
Clinical Trial Results Demonstrate Significant Motor Function Improvement
The CHMP opinion is based on data from the registrational STEER study, along with supportive Phase IIIb STRENGTH and Phase I/II STRONG studies. In the STEER trial, Itvisma (search) demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale (HFMSE) compared to 0.51 points for the sham control group (p=0.0074), with effects sustained over 52 weeks of follow-up.
The STEER and STRENGTH studies showed clinically meaningful benefit for both treatment-naïve and pre-treated patients. Results from the STEER and STRENGTH studies were published in Nature Medicine.
Professor Tim Hagenacker from the Department of Neurology at University Hospital Essen (search), Germany, emphasized the clinical significance of these results: "Itvisma (search) met the primary endpoint showing motor function improvement versus placebo. Even a 1-point difference in the HFSME can translate into tangible functional gains for individuals with SMA (search), such as the ability to grasp a pen. Preserving existing capabilities is critical, as maintaining independence and autonomy is a central goal of care for patients living with a progressive neuromuscular disease."
Addressing Unmet Medical Need in Older SMA Patients
Nicole Gusset, CEO of SMA Europe (search), highlighted the importance of expanding treatment options for older patients: "Living with SMA (search) affects every stage of life, including education, employment, and independence. Older children, teenagers, and adults face fewer opportunities to benefit from innovative therapies. A positive CHMP opinion for Itvisma (search) is an important step toward addressing this gap."
Patrick Horber, MD, President, International, Novartis, noted the potential impact on treatment burden: "Today's positive CHMP opinion is a significant step towards potentially reducing the long-term burden of chronic treatment administration for patients living with SMA (search) in Europe. Building on the established role of Zolgensma for babies and young children with SMA, this opinion for Itvisma (search) reflects our ambition to expand treatment options for a broader patient population in SMA."
Gene Therapy Mechanism and Disease Context
Itvisma (search) is an adeno-associated virus 9 (AAV9)-based gene therapy designed to provide a functional copy of the human SMN1 (search) gene through sustained SMN protein expression with a single, one-time intrathecal injection. By replacing the SMN1 gene, Itvisma can improve motor function and offers the potential to reduce the need for chronically administered treatments associated with other available therapies.
Spinal muscular atrophy (search) is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 (search) gene. The SMN1 gene is responsible for producing most of the SMN protein needed for muscle function, including breathing, swallowing, and basic movement. Without it, motor neurons are irreversibly lost, leading to progressive, debilitating muscle weakness. SMA (search) has an estimated global prevalence of around 1 to 2 per 100,000 people, with an incidence of roughly 1 in 10,000 live births.
Regulatory Timeline and Safety Profile
Following the CHMP's recommendation for approval, the European Commission is expected to issue a final decision within approximately two months. The most frequently reported adverse reactions were similar across the STEER, STRENGTH, and STRONG studies.
In the United States, OAV101B has already been approved under the brand name Itvisma (search). The brand name Itvisma has been conditionally accepted by EMA for the investigational product OAV101B (onasemnogene abeparvovec), but the product has not yet received marketing authorization from the European Commission.