Summary

Overview
Global spinal muscular atrophy treatment market reached US$ XX million in 2022 and is expected to reach US$ XX million by 2030, growing at a CAGR of XX% during the forecast period 2023-2030.
Spinal muscular atrophy (SMA) is a genetic (inherited) neuromuscular disease that causes muscles to become weak. People with SMA lose a specific type of nerve cell in the spinal cord called motor neurons that control muscle movement. Without these motor neurons, muscles don’t receive nerve signals that make muscles move. The word atrophy is a medical term that means smaller. With SMA, certain muscles become smaller and weaker due to lack of use.
There is no cure for spinal muscular atrophy. Treatments depend upon the type of SMA and symptoms. Many people with SMA benefit from physical and occupational therapy and assistive devices, such as orthopedic braces, crutches, walkers and wheelchairs. It mostly affects infants and children but can also develop in adults. Symptoms and prognosis vary depending on SMA type. Gene replacement and disease-modifying therapies offer hope.
According to the Cleveland Clinic Organization, approximately 10,000 to 25,000 children and adults are living with spinal muscular atrophy in the United States. It’s a rare disease that affects one out of 6,000 to 10,000 children.
Market Dynamics: Drivers
Rising adoption of novel therapeutics
The rising adoption of novel therapeutics is expected to drive the market over the forecast period. Therapies for spinal muscular atrophy are designed to interfere with the cellular basis of the disease by modifying pre-mRNA splicing and enhancing the expression of the Survival Motor Neuron (SMN) protein, which is only expressed at low levels in this disorder. Novel therapeutics are developing based on this basis, so there is an increasing demand for novel therapies and there is an increasing adoption of these therapies which leads to better patient outcomes.
For instance, in December 2023, The lab of Yongchao C. Ma, Ph.D., at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important limitations of gene therapy and other current therapies for SMA.
In addition, in December 2023, The Hospital Authority (HA) introduced a gene therapy injection for infants suffering from spinal muscular atrophy (SMA), and it is more effective than traditional treatments for the illness. It said a 10-month-old infant was the first patient in Hong Kong to receive this therapy, called onasemnogene abeparvovec.
Novel therapeutics like Zolgensma have demonstrated remarkable efficacy in clinical trials, often leading to substantial improvements in motor function and quality of life for SMA patients. This has contributed to increased confidence and adoption by healthcare professionals and patients alike. In addition, there is an ongoing clinical trial for the development of more advanced novel therapies for enhanced treatment of the condition.
For instance, on September 14, 2023, Biohaven Ltd., a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, released the completing enrollment in RESILIENT, the Phase 3 pivotal study of taldefgrobep alfa in spinal muscular atrophy (SMA).
Further, the increasing prevalence of spinal muscular atrophy, rising FDA approvals for novel therapies, increasing clinical trials to develop more advanced therapies, increasing awareness about the condition and advancements in the development of novel therapies are the factors expected to drive the market over the forecast period.
Restraints
Factors such as complications and side effects associated with the various therapies, the high cost of the treatment, unmet needs and uncertainties regarding treatment response are the factors expected to hamper the market.
Segment Analysis
The global spinal muscular atrophy treatment market is segmented based on disease type, treatment type, route of administration, end-user and region.
The disease-modifying therapy segment accounted for approximately 45.6% of the spinal muscular atrophy treatment market share
The disease-modifying therapy segment is expected to hold the largest market share over the forecast period. These disease-modifying therapy drugs stimulate the production of SMN protein. Nusinersen (Spinraza) is FDA-approved for both children and adults. The provider injects the drug into the space around the spinal canal. And risdaplam (Evrysdi), helps adults and children older than two months. People take risdaplam daily by mouth (orally).
For instance, on August 7, 2020, PTC Therapeutics, Inc. cleared the U.S. Food and Drug Administration (FDA) approval of Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. Evrysdi showed clinically meaningful improvements in motor function and obtainment of developmental milestones across two trials in patients aged two months and above and across all levels of disease severity, including types 1, 2, and 3 SMA.
In addition, in July 2021, Roche launched Evrysdi (risdiplam), an approved treatment in India for spinal muscular atrophy (SMA) patients. It is available in India within 11 months of the US approval. Since its launch, over 4,000 SMA patients across 50-plus countries have benefitted from Evrysdi. It is approved for the treatment of SMA in adults and children two months of age and older.
Further, their wide availability, acceptability and, ease of application helps to hold the largest market share. Risdiplam (Evrysdi) treatment works to stop the SMN2 genes from disrupting protein production, allowing the protein to reach the nerve cells as needed. The dosage is determined by their weight. Clinical trials showed improved muscle function after 12 months in 41% of those taking it. In addition, their wide adoption also increases the demand for the these medications.
For instance, on August 12, 2021, Chugai Pharmaceutical Co., Ltd. launched Evrysdi Dry Syrup 60 mg for the treatment of spinal muscular atrophy (SMA). This approval is based on the results from the FIREFISH study in babies with symptomatic SMA Type 1 and the SUNFISH study in children and young adults with SMA Type 2 or 3.
Geographical Analysis
North America accounted for approximately 41.4% of the market share
North America region is expected to hold the largest market share over the forecast period owing to the strong presence of major players and increasing research activities for the development of advanced therapies. North America especially the United States is very well-known for its strong presence of major players such as pharmaceutical companies. This presence of major players in the region collaborating and merging with other companies to develop more advanced medications to treat SMA.
For instance, on January 02, 2024, Voyager Therapeutics, Inc., a biotechnology company dedicated to advancing neurogenetic medicines, announced a strategic collaboration and capsid license agreement with Novartis Pharma AG to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER capsids and other intellectual property for the respective diseases.
In addition, there is an increasing research activities for the development of more advanced therapeutics for the better treatment of spinal muscular atrophy also helps to drive the market in the region. As the research activities increase, the therapeutics are developed more precisely by reducing the complications associated with the different types of therapies, resulting in better patient outcomes.
For instance, on March 14, 2022, Biogen Inc. released new data and updates from its SPINRAZA (nusinersen) and spinal muscular atrophy (SMA) research program aimed at improving clinical outcomes for people impacted by the disease, including the ASCEND, RESPOND and NURTURE studies.
Competitive Landscape
The major global players in the spinal muscular atrophy treatment market include Novartis AG, F. Hoffmann-La Roche Ltd, Genentech USA, Inc., Biogen Inc., PTC Therapeutics, Inc., Ionis Pharmaceuticals, Inc., Biohaven Ltd, Voyager Therapeutics, Inc., Catalent, Inc and Pfizer Inc. among others.
COVID-19 Impact Analysis
The COVID-19 pandemic significantly impacted the global spinal muscular atrophy treatment market. The coronavirus disease 2019 (COVID-19) pandemic has resulted in the reorganization of healthcare settings affecting the delivery of clinical care to patients with spinal muscular atrophy (SMA). There is a concern that patients with SMA may be at increased risk of manifesting severe symptoms of COVID-19. Currently approved therapies for SMA improve survival and motor function, however, their delivery requires increased exposure to the health system and a dedicated healthcare team.
Market Segmentation
By Disease Type
• Type 0 SMA (Prenatal SMA)
• Type 1 SMA (Infantile SMA, Werdnig-Hoffmann Disease)
• Type 2 SMA (Intermediate SMA, Dubowitz Disease)
• Type 3 SMA (Juvenile SMA, Kugelberg-Welander Disease)
• Type 4 SMA (Late-Onset SMA, Adults)
By Treatment Type
• Disease-Modifying Therapy
• Gene Replacement Therapy
• Others
By Route of Administration
• Intravenous
• Intrathecal
• Oral
• Others
By End-User
• Hospitals
• Specialty Clinics
• Academic and Research Institutes
• Gene Therapy Centers
• Others
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o U.K.
o France
o Spain
o Italy
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o Australia
o Rest of Asia-Pacific
• Middle East and Africa
Why Purchase the Report?
• To visualize the global spinal muscular atrophy treatment market segmentation based on disease type, treatment type, route of administration, end-user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development
• Excel data sheet with numerous data points of spinal muscular atrophy treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.
The global spinal muscular atrophy treatment market report would provide approximately 69 tables, 72 figures, and 187 Pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

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Table of Contents

1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Disease Type
3.2. Snippet by Treatment Type
3.3. Snippet by Route of Administration
3.4. Snippet by End-User
3.5. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Rising Adoption of Novel Therapeutics
4.1.2. Restraints
4.1.2.1. High Cost of the Treatment
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Industry Analysis
5.1. Porter's Five Force Analysis
5.2. Supply Chain Analysis
5.3. Pricing Analysis
5.4. Regulatory Analysis
5.5. Pipeline Analysis
5.6. Unmet Needs
5.7. PESTEL Analysis
5.8. Patent Analysis
5.9. SWOT Analysis
6. COVID-19 Analysis
6.1. Analysis of COVID-19
6.1.1. Scenario Before COVID
6.1.2. Scenario During COVID
6.1.3. Scenario Post COVID
6.2. Pricing Dynamics Amid COVID-19
6.3. Demand-Supply Spectrum
6.4. Government Initiatives Related to the Market During the Pandemic
6.5. Manufacturers Strategic Initiatives
6.6. Conclusion
7. By Disease Type
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
7.1.2. Market Attractiveness Index, By Disease Type
7.2. Type 0 SMA (Prenatal SMA) *
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Type 1 SMA (Infantile SMA, Werdnig-Hoffmann Disease)
7.4. Type 2 SMA (Intermediate SMA, Dubowitz Disease)
7.5. Type 3 SMA (Juvenile SMA, Kugelberg-Welander Disease)
7.6. Type 4 SMA (Late-Onset SMA, Adults)
8. By Treatment Type
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
8.1.2. Market Attractiveness Index, By Treatment Type
8.2. Disease-Modifying Therapy*
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Gene Replacement Therapy
8.4. Others
9. By Route of Administration
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
9.1.2. Market Attractiveness Index, By Route of Administration
9.2. Intravenous*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Intrathecal
9.4. Oral
9.5. Others
10. By End-User
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
10.1.2. Market Attractiveness Index, By End-User
10.2. Hospitals*
10.2.1. Introduction
10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
10.3. Specialty Clinics
10.4. Academic and Research Institutes
10.5. Gene Therapy Centers
10.6. Others
11. By Region
11.1. Introduction
11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
11.1.2. Market Attractiveness Index, By Region
11.2. North America
11.2.1. Introduction
11.2.2. Key Region-Specific Dynamics
11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.2.7.1. U.S.
11.2.7.2. Canada
11.2.7.3. Mexico
11.3. Europe
11.3.1. Introduction
11.3.2. Key Region-Specific Dynamics
11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.3.7.1. Germany
11.3.7.2. UK
11.3.7.3. France
11.3.7.4. Italy
11.3.7.5. Spain
11.3.7.6. Rest of Europe
11.4. South America
11.4.1. Introduction
11.4.2. Key Region-Specific Dynamics
11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.4.7.1. Brazil
11.4.7.2. Argentina
11.4.7.3. Rest of South America
11.5. Asia-Pacific
11.5.1. Introduction
11.5.2. Key Region-Specific Dynamics
11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
11.5.7.1. China
11.5.7.2. India
11.5.7.3. Japan
11.5.7.4. Australia
11.5.7.5. Rest of Asia-Pacific
11.6. Middle East and Africa
11.6.1. Introduction
11.6.2. Key Region-Specific Dynamics
11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
12. Competitive Landscape
12.1. Competitive Scenario
12.2. Market Positioning/Share Analysis
12.3. Mergers and Acquisitions Analysis
13. Company Profiles
13.1. Novartis AG*
13.1.1. Company Overview
13.1.2. Product Portfolio and Description
13.1.3. Financial Overview
13.1.4. Key Developments
13.2. F. Hoffmann-La Roche Ltd
13.3. Genentech USA, Inc.
13.4. Biogen Inc.
13.5. PTC Therapeutics, Inc.
13.6. Ionis Pharmaceuticals, Inc.
13.7. Biohaven Ltd
13.8. Voyager Therapeutics, Inc.
13.9. Catalent, Inc
13.10. Pfizer Inc.
LIST NOT EXHAUSTIVE
14. Appendix
14.1. About Us and Services
14.2. Contact Us