Spinal Muscular Atrophy Market, by Disease Type (Type I, Type II, Type III, Type IV), by Treatment (Gene Therapy and Drugs), by Age (Infant and Adult), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Global Forecast to 2026

  • Published On : Jul 2018 |
  • Pages : 161 Pages |
  • Format : CMI Insight PPT Format CMI Insight PDF Format

Spinal Muscular Atrophy Market– A Genetic Disorder Affecting Part of the Nervous System

Spinal muscular atrophy (SMA) is a genetic disorder that affects the control of muscle movement. It is caused due to loss of specialized nerve cells, called motor neurons, in the spinal cord and brainstem. The loss of motor neurons leads to weakness and atrophy of muscles used for activities such as sitting up, walking, crawling, and controlling head movement. In severe cases, the muscles used for swallowing and breathing are also affected. There are four types of spinal muscular atrophy, which are differentiated based on the severity of muscle weakness, pattern of features, and age, when the muscle problems begin. These are classified as type 1, 2, and 3 that appears in childhood. Type 4 appears in adulthood and is also termed as adult onset SMA.

The global spinal muscular atrophy market size was valued at US$ 884 million in 2017 and is expected to witness a robust CAGR of 12.4% over the forecast period (2018–2026).

Figure 1. Global Spinal Muscular Atrophy Market Share (%), By Age

 

Source: Coherent Market Insights Analysis (2018)

Increasing number of clinical trials and government funding for R&D is expected to bolster the spinal muscular atrophy market growth

Various government and private organizations are investing huge capital and resources towards the development of new and effective therapies for spinal muscular atrophy. For instance, in 2015, the SMA Foundation financed over US$ 60 Mn in drug development programs and essential drug discovery assets that aim to develop an effective SMA therapy. In 2013, Cure SMA granted fund to Cytokinetics for the study of Tirasemtiv for SMA. Financial details of the grant were not disclosed. Tirasemtiv is a novel skeletal muscle activator and a lead drug candidate from Cytokinetics' skeletal muscle contractility program. It has been studied in clinical trials for individuals with amyotrophic lateral sclerosis (ALS). Similar to SMA, ALS also affects motor neurons.

The National Institute of Neurological Disorders and Stroke (NINDS), a part of the National Institutes of Health (NIH), conducts clinical, translational, and basic research on SMA and also supports research through grants to major medical institutions across the U.S. In 2011, the authority established NeuroNext clinical trials network to promote rapid development and implementation of trials for neurological disorders that affect adults and/or children. The network is designed to develop early-phase trials aimed at identifying biomarkers that can be measured to determine the presence and severity of a disease—and testing promising, emerging therapies.

Figure 2. Spinal Muscular Atrophy Therapy Pipeline, 2017

Increasing incidence rate of spinal muscular atrophy worldwide is expected to augment the market growth

Spinal muscular atrophy mainly manifests early in life and therefore is a major genetic cause of death in infants. It is one of the most common rare diseases. According to SMA Foundation 2015 statistics, spinal muscular atrophy has generally been believed to affect as many as 10,000 to 25,000 adults and children in the U.S. In March 2018, CureSMA stated that SMA affects around 1 in 11,000 children, and 1 in 50 is a carrier of the disease. It has been found in people of every race, however, is most common in Caucasians, of whom 1 in 35 is a carrier. Spinal muscular atrophy carrier rates for other populations include Asians (1 in 53), African Americans (1 in 66), and Hispanics (1 in 117).

However, high cost of therapy can confine growth of the spinal muscular atrophy market during the forecast period. For instance, Biogen’s Spinraza (nusinersen) drug for spinal muscular atrophy is estimated to cost US$ 125,000 per injection, accounting for US$ 750,000 for the first year and US$ 375,000 later.

Major players operating in the global spinal muscular atrophy market include Biogen, Cytokinetics, Inc., F. Hoffmann-La Roche, Novartis AG, Pfizer Inc., and Ionis Pharmaceuticals, Inc.

Spinal muscular atrophy (SMA) is a rare, genetically inherited neuromuscular condition. The disease causes loss of movement and progressive muscle weakness due to muscle wasting (atrophy). The disease is divided into four subtypes (1 to 4) on the basis of the age of onset. SMA type 1 (SMA1) is the most severe form and most common genetic cause of death among infants. Various drugs are available in the market to treat the symptoms of the diseases, however, only one drug is approved for the treatment of SMA so far. Muscle relaxants such as tizanidine, baclofen, and the benzodiazepines can reduce spasticity. Excessive saliva can be treated with glycopyolate, amitriptyline, and atropine or by botulinum injections into the salivary glands. Antidepressants can be helpful in treating depression. Botulinum toxin can also be used to treat drooling or jaw spasms.

Market Dynamics

Major factors driving growth of the spinal muscular atrophy market includes increasing number of clinical trials and rising government support for R&D. Biotechnology and biopharmaceutical companies are working towards development of effective therapies to treat spinal muscular atrophy. For instance, in 2016, the U.S. FDA approved Spinraza (nusinersen) drug from Biogen for the treatment of SMA. Spinraza targets the underlying defect in SMA, so it can help prevent, delay, or even reverse the symptoms.

However, its common side effects include higher risk of constipation and respiratory tract infection along with minimal risk of kidney problems and bleeding. Moreover, the cost of research for development of new therapy is also high. With the advancements of drug in the pipeline, its expense and time for development also increases. According to Cure SMA, it can take over 15 years and US$ 100 million for a single drug candidate to reach FDA approval. These factors make research challenging particularly for a disease such as SMA. These factors are expected to hinder growth of the market.

Key features of the study:

  • This report provides in-depth analysis of spinal muscular atrophy market and provides market size (US$ Million) and Compound Annual Growth Rate (CAGR %) for the forecast period (2018 – 2026), considering 2017 as the base year
  • It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrix for this market
  • This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategy adopted by the leading players
  • It profiles leading players in the global spinal muscular atrophy market based on the following parameters – company overview, financial performance, product portfolio, market presence, key developments, strategies, and future plans
  • Key companies covered as a part of this study include Biogen, Cytokinetics, Inc., F. Hoffmann-La Roche, Novartis AG, Pfizer Inc., and Ionis Pharmaceuticals, Inc.
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launch, technology up-gradation, market expansion, and marketing tactics
  • The global spinal muscular atrophy market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
  • Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the spinal muscular atrophy market

Detailed Segmentation:

  • Global Spinal Muscular Atrophy Market, By Disease Type:
    • Type I
    • Type II
    • Type III
    • Type IV
  • Global Spinal Muscular Atrophy Market, By Treatment:
    • Gene Therapy
    • Drugs
  • Global Spinal Muscular Atrophy Market, By Age:
    • Infant
    • Adult
  • Global Spinal Muscular Atrophy Market, By Geography:
    • North America
      • By Disease Type:
        • Type I
        • Type II
        • Type III
        • Type IV
      • By Treatment:
        • Gene Therapy
        • Drugs
      • By Age:
        • Infant
        • Adult
      • By Country:
        • U.S.
        • Canada
    • Latin America
      • By Disease Type:
        • Type I
        • Type II
        • Type III
        • Type IV
      • By Treatment:
        • Gene Therapy
        • Drugs
      • By Age:
        • Infant
        • Adult
      • By Country:
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • By Disease Type:
        • Type I
        • Type II
        • Type III
        • Type IV
      • By Treatment:
        • Gene Therapy
        • Drugs
      • By Age:
        • Infant
        • Adult
      • By Country:
        • U.K.
        • Germany
        • Italy
        • France
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • By Disease Type:
        • Type I
        • Type II
        • Type III
        • Type IV
      • By Treatment:
        • Gene Therapy
        • Drugs
      • By Age:
        • Infant
        • Adult
      • By Country:
        • China
        • India
        • Japan
        • ASEAN
        • Australia
        • South Korea
        • Rest of Asia Pacific
    • Middle East
      • By Disease Type:
        • Type I
        • Type II
        • Type III
        • Type IV
      • By Treatment:
        • Gene Therapy
        • Drugs
      • By Age:
        • Infant
        • Adult
      • By Country:
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • By Disease Type:
        • Type I
        • Type II
        • Type III
        • Type IV
      • By Treatment:
        • Gene Therapy
        • Drugs
      • By Age:
        • Infant
        • Adult
      • By Country:
        • Central Africa
        • South Africa
        • North Africa
  • Company Profiles
    • Biogen*
      • Company Overview
      • Product Portfolio
      • Key Highlights
      • Financial Overview
      • Market Strategy
    • Cytokinetics, Inc.
    • F. Hoffmann-La Roche
    • Pfizer Inc.
    • Novartis AG
    • Ionis Pharmaceuticals, Inc.

*” marked represents similar segmentation in other categories in the respective section.

Table of Contents

  1. Research Objective and Assumption
    • Research Objectives
    • Assumptions
    • Abbreviations
  2. Market Purview
    • Report Description
      • Market Definition and Scope
    • Executive Summary
      • Market Snippet, By Technology
      • Market Snippet, By Disease Indication
      • Market Snippet, By End User
      • Market Snippet, By Region
    • Coherent Opportunity Map (COM)
  3. Market Dynamics, Regulations, and Trends Analysis
    • Market Dynamics
      • Drivers
      • Restraints
      • Market Opportunities
      • Market Trends
    • Pricing Analysis
    • Clinical Trials For Spinal Muscular Atrophy
    • Regulatory Landscape
    • Reimbursement Scenario
    • Pipeline Analysis
    • Epidemiology
    • PEST Analysis
  4. Global Spinal Muscular Atrophy Market, By Disease Type, 2016–2026, (US$ Million)
    • Introduction
      • Market Share Analysis, 2018 and 2026 (%)
      • Y-o-Y Growth Analysis, 2017–2026
      • Segment Trends
    • Type I
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
    • Type II
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
    • Type III
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
    • Type IV
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
  5.  Global Spinal Muscular Atrophy Market, By Treatment, 2016–2026, (US$ Million)
    • Introduction
      • Market Share Analysis, 2018 and 2026 (%)
      • Y-o-Y Growth Analysis, 2017–2026
      • Segment Trends
    • Gene Therapy
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
    • Drugs
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
  6.  Global Spinal Muscular Atrophy Market, By Age, 2016–2026, (US$ Million)
    • Introduction
      • Market Share Analysis, 2018 and 2026 (%)
      • Y-o-Y Growth Analysis, 2017–2026
      • Segment Trends
    • Infant
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
    • Adult
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Million)
  7.  Global Spinal Muscular Atrophy Market, By Region, 2016 – 2026, (US$ Million)
    • Introduction
      • Market Share Analysis, By Region, 2018 and 2026 (%)
      • Y-o-Y Growth Analysis, For Regions, 2017–2026
    • North America
      • Market Size and Forecast, By Disease Type, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Treatment, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Age, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Country, 2016–2026, (US$ Million)
        • U.S.
        • Canada
    • Latin America
      • Market Size and Forecast, By Disease Type, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Treatment, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Age, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Country, 2016–2026, (US$ Million)
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • Market Size and Forecast, By Disease Type, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Treatment, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Age, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Country, 2016–2026, (US$ Million)
        • Germany
        • U.K.
        • France
        • Italy
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • Market Size and Forecast, By Disease Type, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Treatment, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Age, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Country, 2016–2026, (US$ Million)
        • China
        • India
        • Japan
        • Australia
        • South Korea
        • ASEAN
        • Rest of Asia Pacific
    • Middle East
      • Market Size and Forecast, By Disease Type, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Treatment, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Age, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Country, 2016–2026, (US$ Million)
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • Market Size and Forecast, By Disease Type, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Treatment, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Age, 2016–2026, (US$ Million)
      • Market Size and Forecast, By Country, 2016–2026, (US$ Million)
        • South Africa
        • Central Africa
        • North Africa
  8. Competitive Landscape
    • Heat Map Analysis
    • Company Profiles
      • Biogen*
        • Company Overview
        • Product Portfolio
        • Key Highlights
        • Financial Overview
        • Market Strategy
      • Cytokinetics, Inc.
        • Company Overview
        • Product Portfolio
        • Key Highlights
        • Financial Overview
        • Market Strategy
      • F. Hoffmann-La Roche
        • Company Overview
        • Product Portfolio
        • Key Highlights
        • Financial Overview
        • Market Strategy
      • Pfizer Inc.
        • Company Overview
        • Product Portfolio
        • Key Highlights
        • Financial Overview
        • Market Strategy
      • Novartis AG
        • Company Overview
        • Product Portfolio
        • Key Highlights
        • Financial Overview
        • Market Strategy
      • Ionis Pharmaceuticals, Inc.
        • Company Overview
        • Product Portfolio
        • Key Highlights
        • Financial Overview
        • Market Strategy
    • Analyst Views
  9. Section
    • References
    • Research Methodology
    • About us and Sales Contact

*Browse 32 market data tables and 26 figures on "Spinal Muscular Atrophy Market - Global forecast to 2026”.

Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.


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Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.

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Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization:

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users

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