Spinal Muscular Atrophy Treatment Market to Grow at 18.2% CAGR Through 2032: Persistence Market Research
Spinal Muscular Atrophy (SMA) Treatment Market
Spinal muscular atrophy treatment market driven by gene therapy, antisense drugs, and newborn screening, with strong growth in advanced therapies worldwide.
BRENTFORD, ENGLAND, UNITED KINGDOM, October 8, 2025 /EINPresswire.com/ -- The global spinal muscular atrophy (SMA) treatment market is on a robust growth trajectory, with projections indicating a market value of USD 4.6 billion in 2025, expected to reach USD 14.8 billion by 2032, growing at a compound annual growth rate (CAGR) of 18.2% during the forecast period (2025–2032). This significant expansion is driven by advancements in gene therapy, antisense oligonucleotides, and oral SMN-enhancing drugs, which directly target the genetic cause of SMA, revolutionizing treatment approaches for this debilitating neuromuscular disorder.
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Key Industry Highlights
Leading Product: Onasemnogene Abeparvovec (Zolgensma)
Onasemnogene Abeparvovec, marketed as Zolgensma, is the leading product in the SMA treatment market, due to its unique one-time gene replacement therapy that addresses the root cause of SMA. This therapy has been a game-changer for infants diagnosed with Type 1 SMA, offering a highly effective treatment option that provides significant improvements in motor function and survival rates. Zolgensma has seen widespread adoption, especially in high-income regions where healthcare systems are well-established.
Type 1 SMA Treatment Growth
Type 1 SMA is the most severe form of the disease, characterized by progressive motor neuron degeneration, leading to muscle weakness, respiratory issues, and often early death if untreated. The latest SMA therapies, including gene therapies like Zolgensma and antisense oligonucleotides like Nusinersen, are specifically designed to target this form of the disease, significantly improving life expectancy and quality of life.
Surge in Hospital Pharmacies
Hospital pharmacies play a critical role in SMA treatment, particularly in administering injectable therapies such as Nusinersen and Zolgensma. These drugs require specialized care for safe and effective administration, with hospital pharmacies expected to dominate the distribution channel segment, capturing over 50% of the market share by 2025.
Regional Trends
North America, particularly the United States, is expected to dominate the SMA treatment market, holding a 45% market share in 2025, primarily due to its advanced healthcare infrastructure, early adoption of innovative therapies, and strong reimbursement policies. In contrast, the Asia Pacific region is the fastest-growing, driven by increasing diagnosis rates and the introduction of subsidized treatment programs in countries like China and India.
Market Dynamics
Driver: Advancements in Gene Therapy and Drug Development
The most prominent driver of growth in the SMA treatment market is the groundbreaking progress in gene therapy and drug development. Over 50 clinical trials for SMA therapies are currently active worldwide, with a significant portion dedicated to gene therapies and antisense oligonucleotides. The introduction of Onasemnogene Abeparvovec and Nusinersen has fundamentally changed the treatment landscape, offering effective solutions that address the genetic root causes of SMA. As the number of clinical trials increases, the market for SMA therapies is set to expand even further, with investments in rare disease treatments expected to exceed USD 1.5 billion by 2025.
Restraint: High Treatment Costs
While the advancements in SMA therapies have been monumental, the high cost of treatment remains a significant challenge for market growth. Onasemnogene Abeparvovec, for instance, costs around USD 2.1 million per dose, while Nusinersen comes at a price of USD 750,000 for the first year, with ongoing annual costs of USD 375,000. These costs are prohibitive for many patients, especially in low-income regions or countries with insufficient healthcare infrastructure. Despite patient assistance programs, the affordability of SMA therapies remains a substantial barrier to broader adoption and access, limiting the market's growth potential in underserved regions.
Opportunity: Healthcare Policy Support and Patient Assistance Programs
One of the most promising opportunities for the SMA treatment market lies in the growing support from healthcare policies and patient assistance programs. Governments and healthcare organizations around the world are increasingly focusing on providing subsidies for rare disease treatments, which are essential for expanding access to SMA therapies. In 2025, it is estimated that 30% of SMA patients will benefit from subsidized treatment programs globally. This trend is particularly evident in the U.S. and Europe, where rare disease funding and orphan drug incentives have significantly reduced the financial burden on patients.
Category-wise Analysis
Product Type Insights
In 2025, Onasemnogene Abeparvovec (Zolgensma) is projected to hold a dominant 60% market share, primarily driven by its transformative impact on Type 1 SMA patients. Zolgensma's ability to provide a one-time cure has made it the preferred treatment for severe cases of SMA, particularly in infants. Nusinersen, an antisense oligonucleotide drug, is expected to contribute 15% to the overall market share, gaining traction for its flexible dosing regimen and its efficacy in treating Type 2 SMA.
Disease Type Insights
Type 1 SMA is the most prevalent and severe form, accounting for 55% of the SMA treatment market in 2025. This form of the disease is particularly responsive to gene replacement therapies like Zolgensma. Type 2 SMA, which typically manifests with less severe symptoms, represents around 12% of the market share and is predominantly treated with RNA-based therapies like Nusinersen.
Distribution Channel Insights
Hospital pharmacies are expected to dominate the distribution channel for SMA therapies, holding a market share of over 50% by 2025. This is due to the specialized nature of SMA treatments, particularly gene therapies and injectable drugs. Retail pharmacies are also experiencing growth, particularly in regions where patient assistance programs are making these treatments more accessible.
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Regional Insights
North America
North America will remain the largest market for SMA treatments, with the U.S. accounting for the lion's share due to its well-established healthcare system, high diagnosis rates, and favorable reimbursement policies. The U.S. market is expected to generate over USD 2 billion in sales by 2025. The demand for SMA therapies, especially for Type 1 SMA, is strong, with 70% of patients expected to receive Zolgensma.
Europe
Europe holds a 25% share of the global SMA treatment market, with Germany, the UK, and France leading the charge. Germany, in particular, is seeing increased demand for SMA treatments, especially for antisense oligonucleotide therapies. The UK’s healthcare system continues to adopt patient assistance programs, boosting access to therapies like Nusinersen, while France has experienced a 10% increase in the use of gene therapies for SMA.
Asia Pacific
Asia Pacific is the fastest-growing region for SMA treatments, driven by countries like China, Japan, and India. China is expected to capture 45% of the regional market share in 2025, fueled by significant increases in healthcare investment. India, with its rising diagnosis rates and expanding healthcare infrastructure, is also seeing growth in SMA therapy adoption.
Competitive Landscape
The SMA treatment market is highly competitive, with several biopharmaceutical companies at the forefront of innovation.
Key players:
Novartis
Ionis Pharmaceuticals
PTC Therapeutics
Scholar Rock Inc.
Cytokinetics
Sarepta Therapeutics
NMD Pharma A/S
Astellas Pharma
Pfizer
Chugai Pharmaceutical
Sanofi S.A. (Genzyme)
Catalyst Pharmaceuticals
Regenxbio Inc.
Biohaven Pharmaceutical Holding Co.
Audentes Therapeutics (Astellas Gene Therapies)
BridgeBio Pharma Inc.
Shionogi & Co., Ltd.
Others
Key Industry Developments
In 2025, Biogen Inc. announced the Phase 1 study results of Salanersen (BIIB115/ION306), an antisense oligonucleotide designed for higher potency and once-yearly dosing. Roche also presented new data on its neuromuscular portfolio, including SMA therapies, at the Muscular Dystrophy Association (MDA) conference in Dallas. In 2024, Biogen reported positive data from a pivotal trial of a higher dose regimen of Nusinersen, which showed improved efficacy in treatment-naïve, symptomatic infants with SMA.
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Market Segmentation
By Product Type:
Nusinersen
Onasemnogen Abeparvovec
By Disease Type:
Type 1 SMA
Type 2 SMA
Others
By Distribution Channel:
Hospital Pharmacies
Retail Pharmacies
Others
By Region:
North America
Europe
East Asia
South Asia and Oceania
Latin America
Middle East and Africa
Future Outlook
The global SMA treatment market is poised for significant growth in the coming years. While challenges such as high treatment costs and limited access in low-income regions remain, the increasing support from healthcare policies, growing clinical trials, and the development of more affordable treatment options will pave the way for broader adoption. As the market expands, innovation in gene therapies and antisense oligonucleotide drugs will continue to drive the evolution of SMA treatments, improving outcomes for patients worldwide. With a projected CAGR of 18.2%, the SMA treatment market is on track to become one of the fastest-growing sectors in the biopharmaceutical industry.
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