Global Spinal Muscular Atrophy (SMA) Therapeutics Market Growth, Share, Size, Trends and Forecast (2025 - 2031)

By Treatment;

Gene Therapy and Drug

By Route Administration;

Oral and Intrathecal.

By Geography;

North America, Europe, Asia Pacific, Middle East and Africa and Latin America - Report Timeline (2021 - 2031).
Report ID: Rn430388690 Published Date: January, 2025 Updated Date: February, 2025

Introduction

Global Spinal Muscular Atrophy (SMA) Therapeutics Market (USD Million), 2021 - 2031

In the year 2024, the Global Spinal Muscular Atrophy (SMA) Therapeutics Market was valued at USD 4,473.61 million. The size of this market is expected to increase to USD 20,858.55 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 24.6%.

The global Spinal Muscular Atrophy (SMA) therapeutics market is a dynamic sector within the pharmaceutical industry, dedicated to addressing the needs of individuals affected by this rare genetic disorder. SMA is characterized by progressive muscle weakness and atrophy, resulting from the degeneration of motor neurons in the spinal cord. Historically, SMA had limited treatment options, with supportive care being the primary approach. However, recent years have witnessed a revolution in SMA treatment, driven by significant advancements in genetic research and therapeutic innovation.

Groundbreaking developments in SMA therapeutics is the advent of disease-modifying treatments that target the underlying genetic cause of the condition. Among these, gene replacement therapies, such as onasemnogene abeparvovec, have garnered considerable attention for their ability to address the root cause of SMA by replacing the defective or missing SMN1 gene. Additionally, small molecule therapies, including nusinersen and risdiplam, have emerged as promising alternatives, offering targeted approaches to increase SMN protein levels and improve motor function in individuals with SMA. These innovative therapies have transformed the treatment landscape for SMA, providing hope and improved outcomes for patients and their families.

Challenges persist in the SMA therapeutics market, including access to treatment, affordability, and the need for further research into long-term efficacy and safety. Additionally, as therapeutic options continue to expand, there is a growing emphasis on personalized medicine approaches to optimize treatment outcomes for individuals with SMA. Overall, the global SMA therapeutics market is poised for continued growth and innovation, driven by ongoing research efforts, collaborations between industry stakeholders, and a shared commitment to improving the lives of those affected by this debilitating disorder.

  1. Introduction
    1. Research Objectives and Assumptions
    2. Research Methodology
    3. Abbreviations
  2. Market Definition & Study Scope
  3. Executive Summary
    1. Market Snapshot, By Treatment
    2. Market Snapshot, By Route Administration
    3. Market Snapshot, By Region
  4. Global Spinal Muscular Atrophy (SMA) Therapeutics Market Dynamics
    1. Drivers, Restraints and Opportunities
      1. Drivers
        1. Improved Clinical Trial Designs
        2. Expanded Newborn Screening Programs
        3. Rising Healthcare Investments
        4. Supportive Reimbursement Policies
      2. Restraints
        1. Market Competition
        2. Healthcare Infrastructure Gaps
        3. Insurance Coverage Limitations
        4. Drug Development Risks
      3. Opportunities
        1. Market Expansion in Emerging Economies
        2. Technology Integration
        3. Therapeutic Innovations
        4. Healthcare Policy Changes
    2. PEST Analysis
      1. Political Analysis
      2. Economic Analysis
      3. Social Analysis
      4. Technological Analysis
    3. Porter's Analysis
      1. Bargaining Power of Suppliers
      2. Bargaining Power of Buyers
      3. Threat of Substitutes
      4. Threat of New Entrants
      5. Competitive Rivalry
  5. Market Segmentation
    1. Global Spinal Muscular Atrophy (SMA) Therapeutics Market, By Treatment, 2021 - 2031 (USD Million)
      1. Gene Therapy
      2. Drug
    2. Global Spinal Muscular Atrophy (SMA) Therapeutics Market, By Route Administration, 2021 - 2031 (USD Million)
      1. Oral
      2. Intrathecal
    3. Global Spinal Muscular Atrophy (SMA) Therapeutics Market, By Geography, 2021 - 2031 (USD Million)
      1. North America
        1. United States
        2. Canada
      2. Europe
        1. Germany
        2. United Kingdom
        3. France
        4. Italy
        5. Spain
        6. Nordic
        7. Benelux
        8. Rest of Europe
      3. Asia Pacific
        1. Japan
        2. China
        3. India
        4. Australia & New Zealand
        5. South Korea
        6. ASEAN (Association of South East Asian Countries)
        7. Rest of Asia Pacific
      4. Middle East & Africa
        1. GCC
        2. Israel
        3. South Africa
        4. Rest of Middle East & Africa
      5. Latin America
        1. Brazil
        2. Mexico
        3. Argentina
        4. Rest of Latin America
  6. Competitive Landscape
    1. Company Profiles
      1. Biogen
      2. Ionis Pharmaceuticals
      3. F. Hoffmann
      4. La Roche Ltd
      5. Avexis
      6. Novartis AG
      7. Cytokinetics
  7. Analyst Views
  8. Future Outlook of the Market