Spinal Muscular Atrophy (SMA) Treatment Market Size to Reach US$14.8 Bn by 2032, Growing at 18.2% CAGR - Persistence Market Research

The global spinal muscular atrophy (SMA) treatment market is undergoing significant transformation, fueled by advances in gene therapy and novel drug platforms. Valued at US$4.6 billion in 2025, the industry is projected to surge to US$14.8 billion by 2032, growing at a remarkable CAGR of 18.2% during the forecast period. According to Persistence Market Research, the landscape is increasingly shaped by therapies that target the root cause of SMA, providing long-term solutions for patients across disease types.

Key growth drivers include the adoption of newborn screening programs, rising awareness, and favorable regulatory frameworks that accelerate approvals and reimbursement for high-cost therapies. Gene replacement treatments like Onasemnogene Abeparvovec (Zolgensma) and antisense oligonucleotides such as Nusinersen (Spinraza) dominate the market. Type 1 SMA accounts for the largest disease segment due to its high prevalence and severity, while North America remains the leading geographical region, backed by robust healthcare infrastructure, research funding, and rapid uptake of innovative therapies.

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Key Highlights from the Report

• Onasemnogene Abeparvovec (Zolgensma) leads the market with its one-time gene therapy approach.
• Type 1 SMA treatment demand surges due to early intervention needs in infants.
• Hospital pharmacies hold the largest share due to injectable SMA drug administration.
• North America commands a 45% market share, while Asia Pacific is the fastest-growing.
• Antisense oligonucleotide therapies enhance genetic disorder treatment by 18%.
• Newborn screening programs fuel early diagnosis and expand treatment accessibility.

Market Segmentation

The spinal muscular atrophy treatment market is segmented by product type, disease type, and distribution channel. Onasemnogene Abeparvovec dominates the product landscape with over 60% market share in 2025, thanks to its transformative role as a gene replacement therapy. Nusinersen remains a critical player for Type 2 SMA patients, benefiting from patient assistance programs and flexible dosing regimens.

By disease type, Type 1 SMA represents more than half of the total market share, driven by the high prevalence of this severe form, which accounts for nearly 60% of all SMA cases. Type 2 SMA is also gaining traction as diagnosis rates improve and access to RNA-based therapies increases. Distribution-wise, hospital pharmacies dominate, serving as primary hubs for administering complex injectable therapies, while retail pharmacies are seeing steady growth as they expand access to oral and maintenance-based SMA treatments.

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Regional Insights

In North America, the SMA treatment market thrives on advanced infrastructure, high awareness, and early adoption of breakthrough therapies. The U.S. alone generated more than US$2 billion in SMA treatment sales in 2025, supported by extensive reimbursement and regulatory approvals.

Europe holds a 25% share of the global market, with Germany, the UK, and France leading due to strong healthcare funding and widespread adoption of antisense oligonucleotides. Meanwhile, Asia Pacific emerges as the fastest-growing region, posting a CAGR of nearly 19%. China and India are investing heavily in rare disease funding and newborn screening, making the region a future hotspot for SMA therapy adoption.

Market Drivers

The primary driver of market growth is technological advancement in gene therapy and RNA-based treatments. Over 50 clinical trials were active globally in 2025, with 30% targeting gene therapies and 25% focusing on antisense oligonucleotides. Breakthrough therapies like Zolgensma have delivered survival rates of up to 80% in Type 1 SMA patients, revolutionizing treatment outcomes. Strong FDA and EMA support, orphan drug designations, and patient-centric healthcare policies further accelerate adoption.

Market Restraints

Despite rapid progress, the market faces a significant restraint in the form of exorbitant treatment costs. Zolgensma averages US$2.1 million per dose, while Spinraza can cost upwards of US$750,000 in the first year alone. These price points restrict accessibility in low-income regions and create reimbursement challenges even in developed nations. As a result, treatment delays and inequitable access remain pressing concerns.

Market Opportunities

Expanding healthcare policy support and patient assistance programs present strong opportunities for market expansion. In 2025, over 30% of SMA patients benefited from subsidized treatments globally, with the U.S. and EU allocating nearly US$500 million to rare disease funding. Asia Pacific nations are also rolling out new initiatives to subsidize therapies and improve newborn screening, opening pathways for broader treatment adoption. These supportive measures encourage biopharma investment in R&D and make advanced therapies more accessible.

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Reasons to Buy the Report

• Provides a comprehensive analysis of global and regional SMA treatment trends through 2032.
• Identifies key market segments and emerging therapy classes driving industry transformation.
• Delivers in-depth insights on growth drivers, restraints, and opportunities.
• Offers competitive landscape analysis, including strategies of leading players.
• Highlights regulatory and reimbursement frameworks that shape market access.

Frequently Asked Questions (FAQs)

• How Big is the Spinal Muscular Atrophy (SMA) Treatment Market in 2025?
• Who are the Key Players in the Global SMA Treatment Market?
• What is the Projected Growth Rate of the SMA Treatment Market between 2025 and 2032?
• What is the Market Forecast for SMA Treatments by 2032?
• Which Region is Estimated to Dominate the SMA Treatment Industry through the Forecast Period?

Company Insights

The global SMA treatment market is characterized by strong competition and continuous innovation. Leading companies focus on developing transformative therapies while expanding access through assistance programs.

• Novartis
• Biogen (Ionis Pharmaceuticals collaboration)
• PTC Therapeutics
• Scholar Rock Inc.
• Cytokinetics
• Sarepta Therapeutics
• NMD Pharma A/S
• Astellas Pharma / Audentes Therapeutics
• Pfizer
• Chugai Pharmaceutical
• Sanofi S.A. (Genzyme)
• Catalyst Pharmaceuticals
• Regenxbio Inc.
• Biohaven Pharmaceutical Holding Co.
• BridgeBio Pharma Inc.
• Shionogi & Co., Ltd.

Recent Developments

• In June 2025, Biogen Inc. announced topline results from a Phase 1 trial of salanersen (BIIB115), an advanced antisense oligonucleotide designed for annual dosing in SMA patients.
• In March 2025, Roche presented new data from its neuromuscular portfolio at the MDA conference, reinforcing its pipeline strength in SMA and Duchenne muscular dystrophy.

Conclusion

The spinal muscular atrophy treatment market is set for sustained growth, underpinned by cutting-edge therapies that fundamentally alter patient outcomes. High therapy costs remain a challenge, but rising global policy support and patient assistance initiatives are widening access. With strong investment in R&D and expanding newborn screening programs, the industry is poised to deliver breakthroughs that will reshape the rare disease treatment landscape through 2032.

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