Novartis received the FDA approval for ITVISMA (onasemnogene abeparvovec-brve) on 24th November 2025, marking a significant milestone for patients living with spinal muscular atrophy (SMA) and the broader neurological disease.
This regulatory decision establishes ITVISMA as the first and only gene replacement therapy available for children two years and older, teens, and adults with SMA due to a confirmed mutation in the SMN1 gene. With this approval, Novartis expanded the reach of gene therapy in SMA, targeting a population with historically unmet needs and signaling a transformative advance for genetic medicine.
ITVISMA: First Single-Dose Gene Therapy Targeting the Broad SMA Population
ITVISMA is uniquely developed to address the genetic root cause of Spinal Muscular Atrophy, a rare, autosomal recessive neuromuscular disease caused by mutations or deletions in the SMN1 gene. The disease leads to progressive weakness and loss of motor neurons due to insufficient survival motor neuron (SMN) protein, which is critical for muscle function, breathing, and voluntary movement.
For the first time, eligible patients across age groups can potentially benefit from the therapeutic effects of a one-time, intrathecal dose that delivers a functional copy of the SMN1 gene, driving sustained improvement and stabilization of motor abilities. This approach offers the possibility to significantly reduce the need for chronic therapies that form the current standard of care for Spinal Muscular Atrophy.
ITVISMA’s FDA Approval Supported by Robust Efficacy and Safety Data
ITVISMA’s approval is underpinned by pivotal results from the Phase III STEER study and the open-label Phase IIIb STRENGTH study, demonstrating statistically significant improvements in motor function irrespective of prior Spinal Muscular Atrophy treatment history. Effects were sustained over a 52-week follow-up period, with stabilization of motor abilities that far exceed those typically observed in the historical progression of the disease.
The safety profile of ITVISMA was consistent across trials, with most adverse events characterized as manageable and in line with expectations for gene therapies. The most common events included mild respiratory tract infections, fever, and cold-like symptoms, with minimal discontinuations attributed to therapy-related issues.
Spinal Muscular Atrophy Market Implications and Future Impact
According to DelveInsight market research, approximately 9,000 people in the US alone are living with SMA, and prevalence is anticipated to continue rising as diagnosis and newborn screening improve. The ITVISMA approval is forecast to reshape the therapeutic landscape, expanding access to gene therapy for older patients and adults, and reinforcing Novartis’ market leadership in SMA following earlier gene therapy innovations.
The US spinal muscular atrophy market is expected to grow at a double-digit CAGR due to advancements in diagnosis and treatment, as well as the launch of novel therapies like ITVISMA. By offering a one-time dose gene therapy that does not require body weight adjustment, Novartis addresses key challenges of chronic disease management, potentially reducing the long-term economic burden associated with SMA.
Addressing a Critical Spinal Muscular Atrophy Unmet Need
Despite the availability of other approved therapies such as SPINRAZA (Biogen), EVRYSDI (Roche), and IV Zolgensma, significant gaps remain in treatment strategies to preserve motor function and independence, particularly in older children and adults. The approval of ITVISMA is a direct response to these challenges, expanding the reach of gene therapy to a broader population segment and enhancing patient-centric care.
For more detailed market analysis, epidemiological insights, and unmet needs on the spinal muscular atrophy landscape, download DelveInsight’s comprehensive “Spinal Muscular Atrophy – Market Insights, Epidemiology and Market Forecast” report
Spinal Muscular Atrophy Competitive Landscape: ITVISMA’s Strategic Positioning
The approval of ITVISMA marks a pivotal inflection point in the spinal muscular atrophy treatment landscape, entering a market dominated by three major pharmaceutical players who collectively control over two-thirds of global SMA revenue. Novartis now positions itself alongside Biogen’s SPINRAZA (nusinersen), an established antisense oligonucleotide modifying SMN2 splicing across all phenotypes, and Roche’s EVRYSDI (risdiplam), the first oral SMN2 splicing modifier for pediatric and adult populations. ITVISMA’s differentiation lies in its unique mechanism as the first and only gene replacement therapy approved for patients two years and older, representing a one-time intrathecal intervention that addresses the genetic root cause of SMA rather than managing symptoms through chronic disease-modifying therapy. This positioning extends Novartis’ gene therapy leadership and creates a critical therapeutic option for patients who require or prefer alternatives to chronic treatment regimens, particularly in older children and adult populations previously underserved by gene therapy modalities.
The competitive landscape is rapidly evolving with an expanding clinical pipeline of adjunctive and combination therapies designed to complement baseline SMN-directed treatments. Notable candidates in development include Scholar Rock’s Apitegromab (SRK-015), a selective anti-latent myostatin antibody with planned US launch, Biohaven’s Taldefgrobep Alfa, another myostatin inhibitor in mid-stage development, and emerging programs from Biogen (BIIB115, a longer-acting antisense oligonucleotide), Chugai/Roche (GYM329/RG6237, a muscle-targeting antibody), and NMD Pharma (NMD670, a chloride ion channel inhibitor).
ITVISMA’s approval validates the gene therapy pathway for the broader Spinal Muscular Atrophy patient population and is anticipated to catalyze accelerated clinical development of complementary agents, potentially establishing multi-agent treatment regimens as the emerging standard of care and intensifying competitive pressures across the therapeutic landscape.
For more insights on which companies have the competitive advantage in the Spinal Muscular Atrophy treatment landscape, visit Spinal Muscular Atrophy Competitive Landscape
Strategic Perspective from DelveInsight
The approval of ITVISMA signifies a turning point for the Spinal Muscular Atrophy therapeutics market and underscores the growing potential of gene replacement strategies for managing rare neuromuscular disorders. Pharmaceutical companies and stakeholders should anticipate increased competition, evolving treatment algorithms, and enhanced multidisciplinary care models, all driven by deeper genetic insights and strong clinical evidence.
Conclusion
The FDA approval of ITVISMA represents a transformative milestone in the treatment of spinal muscular atrophy, signifying the maturation of gene replacement therapy as a viable therapeutic modality for a broader patient population than previously accessible through existing therapies. Novartis’ achievement in extending gene therapy eligibility to children two years and older, teens, and adults addresses a critical therapeutic gap in the SMA treatment landscape, where older patients and those requiring alternatives to chronic disease-modifying regimens have historically faced limited options. As the SMA market continues its anticipated double-digit growth trajectory, driven by improving diagnostic infrastructure and newborn screening initiatives, Itvisma is positioned to reshape treatment paradigms and establish multi-agent therapeutic approaches as an emerging standard of care.
For pharmaceutical stakeholders, payers, and healthcare providers, ITVISMA’s approval underscores the accelerating pace of innovation in rare neuromuscular diseases and validates the commercial viability of precision genetic therapies. The competitive landscape will inevitably intensify as pipeline candidates targeting complementary mechanisms advance through clinical development, creating opportunities for differentiated positioning and potential synergistic combinations.
DelveInsight remains committed to providing comprehensive market intelligence, epidemiological insights, and competitive landscape analysis to support strategic decision-making across the evolving SMA therapeutic ecosystem. For pharmaceutical executives seeking to understand market dynamics, patient segmentation, competitive positioning, and commercialization strategies in spinal muscular atrophy, DelveInsight’s Spinal Muscular Atrophy market report offers actionable intelligence to navigate this dynamic and rapidly evolving market opportunity.
