Summary

The 7 major spinal muscular atrophy markets reached a value of US$ 3.0 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 13.0 Billion by 2034, exhibiting a growth rate (CAGR) of 14.28% during 2024-2034.

The spinal muscular atrophy market has been comprehensively analyzed in IMARC's new report titled "Spinal Muscular Atrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Spinal muscular atrophy (SMA) is a rare genetic ailment that affects the motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. It is classified into four different types based on the age of onset and severity of symptoms: types 1, 2, 3, and 4. Some of the common symptoms of spinal muscular atrophy are difficulty with movement, weakness in the limbs, poor muscle tone, delayed motor milestones, joint contractures, etc. Other indications of SMA may include tremors, scoliosis, respiratory problems, and difficulty with swallowing or speaking. The diagnosis of spinal muscular atrophy (SMA) typically involves a combination of clinical evaluation, genetic testing, and imaging studies. In addition to conducting blood tests to check for genetic abnormalities linked to SMA, doctors may perform a physical examination to evaluate muscular strength, tone, and reflexes. Numerous imaging studies, such as magnetic resonance imaging (MRI) or electromyography (EMG), are also used to examine the muscles and nerves. Genetic testing can confirm the presence of SMN1 mutations, identify carriers of the disease, and help determine the type and severity of SMA.

The rising prevalence of rare genetic diseases and the escalating need for developing novel treatment regimens for such ailments are primarily driving the spinal muscular atrophy market. In addition to this, the widespread adoption of medications, including nusinersen and risdiplam, which increase the production of the SMN protein and improve motor function and survival in patients with SMA is also bolstering the market growth. Moreover, the implementation of newborn screening for SMA, which involves testing newborns for the presence of the SMN1 gene, is further propelling the market. Early detection through newborn screening can enable effective treatment and possibly better outcomes for affected children. Besides this, several key players are making extensive investments in research activities for the development of disease-modifying therapies that target the underlying cause of SMA. This, in turn, is acting as another significant growth-inducing factor. Additionally, the increasing utilization of respiratory support, such as non-invasive ventilation and cough assist devices, to help individuals with SMA manage respiratory complications is also creating a positive outlook for the market. Apart from this, the emerging popularity of gene therapy, which improves survival, motor function, and quality of life in patients with SMA type 1, is expected to drive the spinal muscular atrophy market in the coming years.

IMARC Group’s new report provides an exhaustive analysis of the spinal muscular atrophy market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for spinal muscular atrophy and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the spinal muscular atrophy market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the spinal muscular atrophy market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the spinal muscular atrophy market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current spinal muscular atrophy marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the spinal muscular atrophy market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the spinal muscular atrophy market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the spinal muscular atrophy market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of spinal muscular atrophy across the seven major markets?
What is the number of prevalent cases (2018-2034) of spinal muscular atrophy by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of spinal muscular atrophy by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of spinal muscular atrophy by type across the seven major markets?
How many patients are diagnosed (2018-2034) with spinal muscular atrophy across the seven major markets?
What is the size of the spinal muscular atrophy patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of spinal muscular atrophy?
What will be the growth rate of patients across the seven major markets?

Spinal Muscular Atrophy: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for spinal muscular atrophy drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the spinal muscular atrophy market?
What are the key regulatory events related to the spinal muscular atrophy market?
What is the structure of clinical trial landscape by status related to the spinal muscular atrophy market?
What is the structure of clinical trial landscape by phase related to the spinal muscular atrophy market?
What is the structure of clinical trial landscape by route of administration related to the spinal muscular atrophy market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Spinal Muscular Atrophy - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Spinal Muscular Atrophy - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Spinal Muscular Atrophy - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Epidemiology by Type (2018-2034)
7.2.6 Diagnosed Cases (2018-2034)
7.2.7 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Epidemiology by Type (2018-2034)
7.3.6 Diagnosed Cases (2018-2034)
7.3.7 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Epidemiology by Type (2018-2034)
7.4.6 Diagnosed Cases (2018-2034)
7.4.7 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Epidemiology by Type (2018-2034)
7.5.6 Diagnosed Cases (2018-2034)
7.5.7 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Epidemiology by Type (2018-2034)
7.6.6 Diagnosed Cases (2018-2034)
7.6.7 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Epidemiology by Type (2018-2034)
7.7.6 Diagnosed Cases (2018-2034)
7.7.7 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Epidemiology by Type (2018-2034)
7.8.6 Diagnosed Cases (2018-2034)
7.8.7 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Epidemiology by Type (2018-2034)
7.9.6 Diagnosed Cases (2018-2034)
7.9.7 Patient Pool/Treated Cases (2018-2034)

8 Spinal Muscular Atrophy - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Spinal Muscular Atrophy - Unmet Needs

10 Spinal Muscular Atrophy - Key Endpoints of Treatment

11 Spinal Muscular Atrophy - Marketed Products
11.1 List of Spinal Muscular Atrophy Marketed Drugs Across the Top 7 Markets
11.1.1 Evrysdi (Risdiplam) - PTC Therapeutics/Roche
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
11.1.2 Spinraza (Nusinersen) - Biogen/Ionis Pharmaceuticals
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 Sales Across Major Markets
11.1.3 Zolgensma (Onasemnogene abeparvovec) - Novartis Gene Therapies/Nationwide Children's Hospital
11.1.3.1 Drug Overview
11.1.3.2 Mechanism of Action
11.1.3.3 Regulatory Status
11.1.3.4 Clinical Trial Results
11.1.3.5 Sales Across Major Markets

Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 Spinal Muscular Atrophy - Pipeline Drugs
12.1 List of Spinal Muscular Atrophy Pipeline Drugs Across the Top 7 Markets
12.1.1 RG 6237 - Hoffmann-La Roche
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 SRK015 - Scholar Rock
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 ACTX 401 - Alcyone Therapeutics
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Spinal Muscular Atrophy - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Spinal Muscular Atrophy – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Spinal Muscular Atrophy - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Spinal Muscular Atrophy - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Spinal Muscular Atrophy - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Spinal Muscular Atrophy - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Spinal Muscular Atrophy - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Spinal Muscular Atrophy - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Spinal Muscular Atrophy - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Spinal Muscular Atrophy - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Spinal Muscular Atrophy - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Spinal Muscular Atrophy - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Spinal Muscular Atrophy - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Spinal Muscular Atrophy - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Spinal Muscular Atrophy - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Spinal Muscular Atrophy - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Spinal Muscular Atrophy - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Spinal Muscular Atrophy - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Spinal Muscular Atrophy - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Spinal Muscular Atrophy - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Spinal Muscular Atrophy - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Spinal Muscular Atrophy - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Spinal Muscular Atrophy - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Spinal Muscular Atrophy - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Spinal Muscular Atrophy - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Spinal Muscular Atrophy - Access and Reimbursement Overview

16 Spinal Muscular Atrophy - Recent Events and Inputs From Key Opinion Leaders

17 Spinal Muscular Atrophy Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Spinal Muscular Atrophy Market – Strategic Recommendations

19 Appendix