Spinal Muscular Atrophy Treatment Market Size, Share, Growth, Trends, and Global Industry Analysis: By Type (Type 1, Type 2, Type 3, Type 4), By Treatment (Gene Therapy, Drug), By Route of Administration (Oral, Injection), and Region Forecast 2020-2031

Spinal Muscular Atrophy Treatment Market size was valued at US$ 2,300 million in 2024 and is expected to reach US$ 4,194 million by 2031, growing at a significant CAGR of 13.5% from 2025-2031. Moreover, the U.S. Spinal Muscular Atrophy Treatment Market is projected to grow significantly, reaching an estimated value of US$ 950 million by 2031. Spinal muscular atrophy (SMA), a rare but life-threatening genetic condition, has long posed a clinical challenge due to its progressive neuromuscular degeneration and early onset. Over the past decade, the global SMA treatment market has undergone a paradigm shift with the introduction of disease-modifying therapies and targeted gene treatments.

As healthcare systems increase their focus on rare disorders and newborn screening becomes more routine in several countries, awareness and early diagnosis are enabling timely interventions. Regulatory support for orphan drugs and breakthroughs in antisense oligonucleotide and gene therapy platforms have improved the therapeutic landscape. The market is transitioning from palliative to curative intent, opening significant commercial and clinical opportunities for drug developers and care networks worldwide.  Innovations in precision medicine, coupled with favorable reimbursement frameworks in developed economies, are steadily redefining SMA patient outcomes. While previously limited to supportive care, treatment options now include targeted therapies that restore or replace SMN protein function.

Moreover, partnerships between biopharma companies and academic institutes are fostering a robust R&D ecosystem, fueling both first-in-class and next-generation therapies. Market access is improving in middle-income economies through global health collaborations and regulatory harmonization. However, the high treatment cost, particularly for gene therapy, continues to challenge equitable access. Despite this, the SMA treatment market reflects a compelling blend of unmet need, evolving science, and promising commercial potential across geographies.

Facts & Figures

• Approximately 1 in 10,000 live births globally is affected by SMA, underscoring the urgency for effective treatments.
• Over 20 clinical-stage pipeline drugs are currently in development across multiple therapeutic classes for SMA.
• In the U.S., SMA has been included in all 50 states' newborn screening programs, improving early detection rates.
• The launch of gene therapy-based treatments has reduced SMA Type 1 infant mortality significantly in treated populations.

Recent Developments:

• In June 2025, Biogen announced positive Phase1 results for salanersen, a once a year injection post Zolgensma therapy, prompting a direct move to Phase3 trials
• In June 2025, A 16 month old in eastern India became the first child in that region to receive Zolgensma via crowdfunding and the manufacturer’s early access programme, highlighting real world outreach of SMA gene therapy
• In November 2024, Biohaven updated its development program for taldefgrobep alfa, a treatment for spinal muscular atrophy. The RESILIENT spinal muscular atrophy study indicated that while taldefgrobep alfa showed clinically meaningful improvements in motor function on the Motor Function Measurement-32 scale (MFM-32), it did not statistically separate from the placebo group at the primary outcome after 48 weeks.

Spinal Muscular Atrophy Treatment Market Segmentation:

Based on the type:

• Type 1
• Type 2
• Type 3
• Type 4

Type 1 spinal muscular atrophy, also known as Werdnig-Hoffmann disease, accounts for the majority of diagnosed SMA cases and remains the most critical focus in treatment development. Its early onset and severe progression often result in fatal outcomes within the first two years if untreated. The emergence of gene therapy and antisense oligonucleotide treatments has shifted the treatment paradigm for Type 1 patients, offering significantly improved motor function and survival outcomes. Due to its severity, regulatory agencies have prioritized this subtype for fast-track approvals and newborn screening programs. Consequently, Type 1 continues to dominate both the clinical attention and commercial share in the SMA treatment market globally.

Based on the treatment:

• Gene Therapy
• Drug
  • Spinraza
  • Zolgensma (AVXS-101)
  • Evrysdi
  • Others

Gene therapy represents the most transformative development in the SMA treatment landscape and leads the market by value and impact. Its one-time dosing model provides long-term benefits by directly replacing or supplementing the missing SMN1 gene. Therapies such as Zolgensma have demonstrated remarkable improvements in motor milestones, particularly in infants diagnosed early. The high efficacy and potential for lifelong results have made gene therapy a preferred option in severe SMA types, especially Type 1. Although accessibility remains limited by cost and infrastructure requirements, ongoing efforts in local manufacturing, insurance alignment, and regulatory acceleration continue to expand its reach, cementing gene therapy’s leading role in SMA management.

Based on the route of administration:

• Oral
• Injection

Injection remains the most effective and commonly used route of administration for SMA therapies, especially those requiring direct delivery to the central nervous system or bloodstream. Spinraza, an antisense oligonucleotide therapy, is administered intrathecally, targeting the spinal cord to promote SMN2 gene splicing. Similarly, gene therapies are delivered intravenously to ensure systemic correction of the genetic defect. This mode of administration ensures precise drug delivery and rapid therapeutic action in patients with severe disease forms. While oral therapies are emerging, injectables are currently better suited for achieving high efficacy in acute and early-stage SMA, thereby maintaining their dominance in treatment protocols and market share.