Austin, July 25, 2025 (GLOBE NEWSWIRE) -- Spinal Muscular Atrophy Treatment Market Size & Trends:
According to SNS Insider, The Spinal Muscular Atrophy Treatment Market was estimated at USD 4.27 billion in 2024 and is expected to reach USD 16.28 billion by 2032, at a CAGR of 17.87% during the forecast period of 2025-2032.
The global spinal muscular atrophy treatment market is bolstered by awareness, early diagnosis through newborn screening, and evolved gene and RNA-based treatments. Continuous R&D and regulatory encouragement also lead to the growth of the market. While the cost of treatment may be high, the need for new, disease-modifying treatments continues to grow, especially in developed parts of the world with broad-based healthcare access and reimbursement systems.
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The U.S. spinal muscular atrophy treatment market was estimated at USD 1.33 billion in 2024 and is expected to reach USD 4.96 billion by 2032, at a CAGR of 17.49% during the forecast period of 2025-2032
The North American spinal muscular atrophy treatment market is held by the U.S. due to advanced healthcare infrastructure, newborn screening mandates in place, and a robust clinical pipeline. That dominance has been bolstered further by routine identification and the swift uptake of products such as Spinraza, Zolgensma, and Evrysdi, as well as strong payment paradigms and a broad clinical trial landscape.
Key Highlights of the Report:
Type 1 SMA Dominates: Type 1 segment dominated the market in 2024 on account of the early onset of the disease, rapid progress of the disease, and high demand for treatment. Type 1 SMA is usually present by 6 months of age and often results in early diagnosis, earlier treatment, which is rather more effective, and has the capability of improving survival and motor function. Availability and demonstrated efficacy of disease-modifying therapies, including gene replacement and SMN2-targeted treatments, have driven implementation.
SMN2 Splicing Modifiers Lead Treatment Modality: SMN2 splicing modifiers segment dominated in 2024, due to their proven clinical effectiveness, easy delivery, and multiple approvals from regulatory bodies. Specifically, drugs such as nusinersen and risdiplam have been shown to upregulate SMN protein levels by modifying SMN2 gene splicing and improve motor function/survival, especially in pediatric patients. Compared to gene therapy, these therapeutic approaches are more feasible, as they are commonly delivered orally or intrathecal, so they are better suited for long-term treatment.
Intrathecal Route of Administration Prevails: The intrathecal segment held the major share of the spinal muscular atrophy treatment market in 2024, which is attributed to the prevalent use of drugs such as nusinersen (Spinraza). Introduction of the viral vector into the intrathecal space permits direct access to the CNS and can achieve a greater therapeutic benefit when motor neurons are targeted. This formulation may be especially beneficial for pediatric patients, in whom systemic delivery may be less efficacious.
Asia Pacific is Projected to be the Rapidly Expanding Region in the Spinal Muscular Atrophy Treatment Market
The spinal muscular atrophy treatment market, growing with an 18.46% CAGR over the forecast period, owing to the increasing awareness among people, healthcare infrastructure, and the adoption rate of genetic screening and diagnostic programs. Newborn screening is being added to some countries, greatly facilitating earlier diagnosis and treatment, such as in China, Japan, and others. The growing burden of neuromuscular disorders and increasing drug accessibility in emerging economies are also driving treatment adoption and expanding the market.
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Key Spinal Muscular Atrophy Treatment Companies Profiled in the Report
- Biogen Inc.
- Novartis AG
- Roche Holding AG
- PTC Therapeutics Inc.
- Ionis Pharmaceuticals Inc.
- REGENXBIO Inc.
- Scholar Rock Holding Corporation
- Astellas Pharma Inc.
- AveXis Inc.
- Cure SMA
- Audentes Therapeutics
- Biohaven Ltd.
- Catalyst Pharmaceuticals Inc.
- Marinus Pharmaceuticals Inc.
- Sarepta Therapeutics Inc.
- Pfizer Inc.
- Ultragenyx Pharmaceutical Inc.
- Voyager Therapeutics Inc.
- NantKwest
- Cytokinetics Inc. and other players.
Spinal Muscular Atrophy Treatment Market Segmentation
By Disease Type
- Type 0
- Type 1
- Type 2
- Type 3
- Type 4
By Treatment Modality
- Gene Replacement Therapy
- SMN2 Splicing Modifiers
- Antisense-Oligonucleotide Therapy
- Muscle-Directed / Myostatin Inhibitors
- Supportive & Assistive Therapies
By Route of Administration
- Oral
- Intrathecal
- Intravenous
By Age Group
- Neonates (0–6 mo)
- Pediatrics (6 mo–12 y)
- Adolescents (12–18 y)
- Adults (>18 y)
By Distribution Channel
- Hospital Pharmacies
- Specialty Clinics
- Online & Retail Pharmacies
Spinal Muscular Atrophy Treatment Market Report Scope
Report Attributes | Details |
Market Size in 2024 | US$ 4.27 billion |
Market Size by 2032 | US$ 16.28 billion |
CAGR | CAGR of 17.87% From 2024 to 2032 |
Base Year | 2024 |
Forecast Period | 2025-2032 |
Historical Data | 2021-2023 |
Regional Analysis | North America (US, Canada, Mexico), Europe (Germany, France, UK, Italy, Spain, Poland, Turkey, Rest of Europe), Asia Pacific (China, India, Japan, South Korea, Singapore, Australia, Rest of Asia Pacific), Middle East & Africa (UAE, Saudi Arabia, Qatar, South Africa, Rest of Middle East & Africa), Latin America (Brazil, Argentina, Rest of Latin America) |
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Table of Contents – Major Key Points
1. Introduction
2. Executive Summary
3. Market Overview
4. Statistical Insights & Trends Reporting
5. Spinal Muscular Atrophy Treatment Market Segmental Analysis & Forecast, By Disease Type
6. Spinal Muscular Atrophy Treatment Market Segmental Analysis & Forecast, By Treatment Modality
7. Spinal Muscular Atrophy Treatment Market Segmental Analysis & Forecast, By Route of Administration
8. Spinal Muscular Atrophy Treatment Market Segmental Analysis & Forecast, By Age Group
9. Spinal Muscular Atrophy Treatment Market Segmental Analysis & Forecast, By Distribution Channel
10. Spinal Muscular Atrophy Treatment Market Segmental Analysis & Forecast, By Region
11. Competitive Landscape
12. Analyst Recommendations
13. Assumptions
14. Disclaimer
15. Appendix
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