Spinal Muscular Atrophy Market Expected to Experience Accelerated Growth and Market Evolution by 2034 - DelveInsight

DelveInsiht's "Spinal Muscular Atrophy Market Insights, Epidemiology, and Market Forecast-2034′′ report offers an in-depth understanding of the Spinal Muscular Atrophy, historical and forecasted epidemiology as well as the Spinal Muscular Atrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Some of the key facts of the Spinal Muscular Atrophy Market Report:
• The Spinal Muscular Atrophy market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
• In July 2026, Novartis announced that the European Commission (EC) has approved Itvisma® (onasemnogene abeparvovec) for the treatment of 5q spinal muscular atrophy (SMA) in children aged two years and older, adolescents, and adults with a biallelic mutation in the survival motor neuron 1 (SMN1) gene.
• In June 2026, SPINRAZA (nusinersen) is approved for the treatment of spinal muscular atrophy (SMA) in both pediatric and adult patients. The therapy was initially developed by Ionis Pharmaceuticals, a U.S.-based biotechnology company, which transferred the worldwide rights for its development, manufacturing, and commercialization to Biogen in August 2016. SPINRAZA is supplied as a clear, colorless intrathecal injection in a single-dose vial containing 12 mg/5 mL (2.4 mg/mL) of nusinersen.
• In June 2026, Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen for the treatment of spinal muscular atrophy (SMA). This designation is intended to accelerate the development and regulatory review of therapies for serious conditions when early clinical evidence suggests a meaningful improvement over existing treatment options on clinically important endpoints. Salanersen is an investigational next-generation antisense oligonucleotide (ASO) designed for SMA and may offer robust therapeutic efficacy with the convenience of once-yearly dosing.
• In May 2026, The FDA's approval of a higher-dose regimen of nusinersen (Spinraza; Biogen) for spinal muscular atrophy (SMA) in March 2026 represents another significant milestone in the evolution of SMA treatment. Since the initial approval of nusinersen at a 12 mg dose in December 2016, the therapy has transformed the disease's clinical trajectory. The later approvals of onasemnogene abeparvovec-xioi (Zolgensma; Novartis) in 2019 and risdiplam (Evrysdi; Genentech) in 2020 established a three-treatment landscape that has substantially influenced patient care, expanded newborn screening efforts, and improved long-term outcomes and expectations across the full spectrum of SMA severity.
• In December 2025, The Emirates Drug Establishment (EDE) has approved ItvismaTM (onasemnogene abeparvovec), a gene therapy using an adeno-associated viral vector to treat spinal muscular atrophy (SMA) in eligible adults and children aged two years and older. With this approval, the UAE becomes the second country worldwide to authorize this therapy, highlighting its commitment to accelerating patient access to advanced medical innovations. Itvisma addresses the genetic root of SMA by replacing the defective gene, improving motor function and reducing dependence on long-term treatments.
• In June 2025, Roche announced that the European Commission (EC) has approved a label extension for Evrysdi (risdiplam), introducing a room-temperature stable 5mg tablet for spinal muscular atrophy (SMA). The 6.5mm tablet can be swallowed whole or dispersed in water, taken with or without food, and stored without refrigeration. Designed for home administration, Evrysdi remains a convenient, non-invasive, disease-modifying therapy for individuals with SMA.
• In June 2025, Biogen Inc. (Nasdaq: BIIB) announced topline data from its Phase 1 study of salanersen (BIIB115/ION306), an investigational antisense oligonucleotide (ASO) for spinal muscular atrophy (SMA). Developed to provide greater potency than SPINRAZA (nusinersen) while working through the same mechanism, salanersen is intended to offer strong efficacy with a single annual dose. Interim results, which also included SMA patients with prior gene therapy, support advancement into registrational studies. Both 40 mg and 80 mg once-yearly doses were well-tolerated and showed significant neuroprotection, evidenced by neurofilament decline and promising functional gains, including attainment of new WHO milestones.
• In March 2025, Novartis' investigational therapy OAV101 IT, an intrathecal version of the approved gene therapy onasemnogene abeparvovec (Zolgensma), has demonstrated encouraging outcomes in improving patient scores on the Hammersmith Functional Motor Scale Expanded (HFMSE). The results, from the Phase 3 STEER trial (NCT05089656), were recently shared at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
• In March 2025, Novartis announced favorable efficacy and safety results from its Phase III clinical program evaluating OAV101 IT (onasemnogene abeparvovec), an investigational intrathecal therapy for spinal muscular atrophy (SMA) in patients aged two to under 18 years. In the pivotal Phase III STEER trial, treatment with OAV101 IT led to a 2.39-point increase on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to a 0.51-point gain in the sham control group highlighting a significant improvement in motor function specific to SMA.
• In March 2025, Scholar Rock's spinal muscular atrophy (SMA) treatment demonstrated improvements in muscle function among children under 12 in a key clinical study, yet the results failed to excite investors. The Phase III SAPPHIRE trial (NCT05156320) assessed the safety and effectiveness of apitegromab, a monoclonal antibody designed to enhance muscle growth by targeting and inhibiting the inactive forms of myostatin specifically within skeletal muscle.
• In February 2025, A pilot clinical trial suggested that spinal cord stimulation (SCS) may help restore function in patients with spinal muscular atrophy (SMA) by gradually activating dormant motor neurons and enhancing leg muscle strength. Conducted by the University of Pittsburgh School of Medicine (NCT05430113), the study evaluated Medtronic's Vectris SureScan device in three SMA patients. All participants demonstrated improved mobility, each increasing their distance in the six-minute walk test (6MWT) a key indicator of muscle endurance by at least 20 meters after three months of treatment
• In January 2025, Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing novel therapies for spinal muscular atrophy (SMA), cardiometabolic conditions, and other serious diseases involving protein growth factors, has announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab. This muscle-directed therapy is intended to deliver significant motor function improvement in individuals with SMA who are already receiving SMN-targeted treatments. The company also plans to submit a Marketing Authorisation Application to the European Medicines Agency in the first quarter of 2025.
• In January 2025, Analytics firm GlobalData has expressed a positive outlook on the future of Novartis' gene therapy for spinal muscular atrophy (SMA), onasemnogene abeparvovec (OAV101 IT). The therapy may soon be accessible to a wider patient group, thanks to encouraging results from Novartis' Phase III STEER trial (NCT05089656), in which the intrathecal version of the treatment successfully met its primary endpoint.
• Among the three FDA-approved treatments, SPINRAZA and ZOLGENSMA are leading the market, with SPINRAZA securing a notable market share across the 7MM. These innovative therapies have revolutionized the treatment of Spinal Muscular Atrophy and delivered substantial financial gains for the pharmaceutical companies responsible for their creation.
• The classical forms of Spinal Muscular Atrophy are caused by biallelic mutations in the survival motor neuron 1 (SMN1) gene on chromosome 5, leading to a deficiency in SMN proteins. A second gene, SMN2, also generates SMN proteins, but they are typically truncated, with only 10%-20% remaining functional.
• Approximately 60% of newborns with Spinal Muscular Atrophy are diagnosed with type I, while types II and III account for the remaining 40%.
• Key Spinal Muscular Atrophy Companies: Genentech, F. Hoffmann-La Roche, Scholar Rock, Biohaven/ Bristol-Myers Squibb, NMD Pharma, Novartis, Hoffmann-La Roche, Genentech, Inc., Scholar Rock, Roche, Cytokinetics, Biogen, Biohaven Pharmaceuticals, and others
• Key Spinal Muscular Atrophy Therapies: ZOLGENSMA (onasemnogene abeparvovac-xioi), EVRYSDI (risdiplam), SPINRAZA, Apitegromab (SRK-015), Taldefgrobep Alfa (BHV-2000), GYM329/RG6237, NMD670, branaplam, RO7204239, Risdiplam, Apitegromab, GYM329, Reldesemtiv, Nusinersen, taldefgrobep alfa, and others
• The Spinal Muscular Atrophy epidemiology based on gender analyzed that it can be concluded that Spinal Muscular Atrophy (SMA) occurs equally in men and women
• The Spinal Muscular Atrophy market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Spinal Muscular Atrophy pipeline products will significantly revolutionize the Spinal Muscular Atrophy market dynamics.

Spinal Muscular Atrophy Overview
Spinal muscular atrophy (SMA) is an autosomal recessive rare neuromuscular disorder that is characterized by progressive muscle wasting because of the loss of motor neurons in the spinal cord (anterior horn), which controls voluntary muscle movement. Spinal Muscular Atrophy is one of the most common causes of infant death and mobility impairment. It mostly affects proximal muscles and respiratory muscles. The most common form of SMA is known as '5q SMA' due to its genetic cause

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Spinal Muscular Atrophy Epidemiology
The Spinal Muscular Atrophy epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Spinal Muscular Atrophy Epidemiology Segmentation:
The Spinal Muscular Atrophy market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Total Prevalence of Spinal Muscular Atrophy
• Prevalent Cases of Spinal Muscular Atrophy by severity
• Gender-specific Prevalence of Spinal Muscular Atrophy
• Diagnosed Cases of Episodic and Chronic Spinal Muscular Atrophy

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Spinal Muscular Atrophy Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Spinal Muscular Atrophy market or expected to get launched during the study period. The analysis covers Spinal Muscular Atrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Spinal Muscular Atrophy Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Spinal Muscular Atrophy Therapies and Key Companies
• ZOLGENSMA (onasemnogene abeparvovac-xioi): Novartis
• EVRYSDI (risdiplam): Genentech/ F. Hoffmann-La Roche
• SPINRAZA: Biogen
• Apitegromab (SRK-015): Scholar Rock
• Taldefgrobep Alfa (BHV-2000): Biohaven/ Bristol-Myers Squibb
• GYM329/RG6237: Roche
• NMD670: NMD Pharma
• branaplam: Novartis
• RO7204239: Hoffmann-La Roche
• Risdiplam: Genentech, Inc.
• Apitegromab: Scholar Rock
• GYM329: Roche
• Reldesemtiv: Cytokinetics
• Nusinersen: Biogen
• taldefgrobep alfa: Biohaven Pharmaceuticals

Discover more about therapies set to grab major Spinal Muscular Atrophy market share @ Spinal Muscular Atrophy Treatment Market
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Spinal Muscular Atrophy Market Drivers
• Newborn Screening for Spinal Muscular Atrophy
• Promising Upcoming Launches and Approval
• Raising Awareness for Spinal Muscular Atrophy

Spinal Muscular Atrophy Market Barriers
• High Spinal Muscular Atrophy treatment Cost
• Competition for Spinal Muscular Atrophy Emerging Therapies

Scope of the Spinal Muscular Atrophy Market Report
• Study Period: 2020-2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Spinal Muscular Atrophy Companies: Genentech, F. Hoffmann-La Roche, Scholar Rock, Biohaven/ Bristol-Myers Squibb, NMD Pharma, Novartis, Hoffmann-La Roche, Genentech, Inc., Scholar Rock, Roche, Cytokinetics, Biogen, Biohaven Pharmaceuticals, and others
• Key Spinal Muscular Atrophy Therapies: ZOLGENSMA (onasemnogene abeparvovac-xioi), EVRYSDI (risdiplam), SPINRAZA, Apitegromab (SRK-015), Taldefgrobep Alfa (BHV-2000), GYM329/RG6237, NMD670, branaplam, RO7204239, Risdiplam, Apitegromab, GYM329, Reldesemtiv, Nusinersen, taldefgrobep alfa, and others
• Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies
• Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Spinal Muscular Atrophy Unmet Needs, KOL's views, Analyst's views, Spinal Muscular Atrophy Market Access and Reimbursement

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