CNS Specific Antisense Oligonucleotide Market - by Drug (Approved and Pipeline), by Indication (Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy, Spinal Muscular Atrophy, and Huntington’s Disease), by Distribution Channel (Hospital Pharmacy, Retail Pharmacy, and Online Pharmacy), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2018 – 2026
- Report code : CMI62477 |
- Industry :
Pharmaceutical
- Published On : Feb 2019 |
- Pages : 206 Pages |
- Format :
Global CNS Specific Antisense Oligonucleotide Market – Breakthrough Treatment for CNS Disorders
Antisense oligonucleotide plays a major role in modern healthcare, as it acts on the messenger RNA level before the protein is formed to inhibit expression of certain diseases-causing genes.
The global CNS specific antisense oligonucleotide market size was valued at US$ 883.7 million in 2017, and is expected to exhibit a CAGR of 24.9% over the forecast period (2018 – 2026).
Figure 1. Global CNS Specific Antisense Oligonucleotide Market Value (US$ Mn), by Region, 2017
Source: Coherent Market Insights Analysis (2019)
Increasing prevalence of cancer is expected to propel growth of the CNS specific antisense oligonucleotide market
CNS specific antisense oligonucleotide is used for treatment of neurodegenerative disorders and associated rare diseases. According to February 2017, report of National Center for Biotechnology Information (NCBI), an estimated the prevalence of Cerebral palsy ranges from 1.5 to more than 4 per 1,000 of children from age 10 to 14 years. The birth prevalence of Cerebral palsy is around 2 per 1,000 live births globally.
Furthermore, launch of new products for rare disease associated with CNS disorders is propelling growth of the CNS specific antisense oligonucleotide market over the forecast period. For instance, in October 2018, Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received the U.S. Food and Drug Administration (FDA) approval for its novel drug TEGSEDITM (inotersen) indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. In the same year, company received approval from Canadian Health authority for the novel drug TEGSEDITM (inotersen).
However, high cost of treatment and risk of toxicity is expected to hinder the market growth. According to the Journal Molecular Therapy, in May 2017, Biogen priced Spinraza (nusinersen injection) at US$ 750,000 for the first year’s treatment (US$125,000 per injection) and US$350,000 per year.
Figure 2. Global CNS Specific Antisense Oligonucleotide Market Share (%), by Drug, 2018 and 2026
Source: Coherent Market Insights Analysis (2019)
Government support for development and launch of antisense drugs in North America is expected to bolster the market growth
North America holds the dominant position in the global CNS specific antisense oligonucleotide market, owing to strong government support for antisense drugs. For instance, in March 2018, GeneTx Biotherapeutics LLC’s, GTX-101 received the U.S. Food and Drug Administration’s (FDA) orphan-drug designation for GTX-101 for the treatment of Angelman syndrome. It is a rare neurogenetic disorder that affects around one in 15,000 people, according to the FDA statement.
Increasing prevalence of Alzheimer disease in the region is also expected to boost the market growth. For instance, in August 2012, National Center for Biotechnology Information (NCBI) stated Alzheimer disease as the most frequent cause of dementia in Western societies and estimated around 5.5 million cases in the U.S. and the prevalence worldwide is estimated to be as high as 24 million.
Moreover, rich product pipeline for treatment of neurodegenerative disorders is expected to drive the market growth. For instance, in October 2017, Ionis Pharmaceuticals, Inc., initiated a Phase 1/2 clinical study of IONIS-MAPTRx in patients with mild Alzheimer's disease. Biogen funded US$ 10 million milestone payment to Ionis Pharmaceuticals, Inc.
Moreover, key players in the players are focused on receiving the U.S. Food and Drug Administration (FDA) approval for novel products to reduce the burden of CNS associated disorders. For instance, in September 2016, Sarepta Therapeutics received the U.S. Food and Drug Administration (FDA) approval for Exondys 51(eteplirsen) used for treatment of rare genetic disorder Duchenne muscular dystrophy.
Key Players
Major players operating in the global CNS specific antisense oligonucleotide market include Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., and Q-STATE BIOSCIENCES, INC.
Antisense oligonucleotides are synthetic strings of nucleic acid, which reduce expression of messenger RNA (m-RNA). The function performed by interference with pre m- RNA splicing are restoration of protein and modification of protein. Antisense oligonucleotide is a new approach to treat several neurodegenerative disorders to prevent disease onset or stop disease development.
Market Dynamics
Increasing prevalence of neurodegenerative disease is a major factor driving CNS specific antisense oligonucleotide market growth. According to fact sheet from Alzheimer Association, an estimated around 5.7 million Americans were diagnosed with Alzheimer's dementia in 2018. Moreover, rich product pipeline for the treatment of CNS disorders and rare diseases associated with it is expected to boost the market growth. For instance, in December 2018, Biogen and Ionis Pharmaceuticals product IONIS-SOD1Rx (BIIB067) cleared Phase 1 clinical trial and is expected to be launched by 2020.
Key features of the study:
- This report provides in-depth analysis of the CNS specific antisense oligonucleotide 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 matrices 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 key players
- It profiles key players in the global CNS specific antisense oligonucleotide market based on the following parameters – company overview, financial performance, product portfolio, market presence, distribution strategies, key developments, strategies, and future plans
- Key companies covered as a part of this study include, Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., Q-STATE BIOSCIENCES, INC.
- Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, technology up-gradation, market expansion, and marketing tactics
- The global CNS specific antisense oligonucleotide 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 CNS specific antisense oligonucleotide market
Detailed Segmentation:
- Global CNS Specific Antisense Oligonucleotide Market, By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- Global CNS Specific Antisense Oligonucleotide Market, By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- Global CNS Specific Antisense Oligonucleotide Market, By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- Global CNS Specific Antisense Oligonucleotide Market, By Geography:
- North America
- By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- By Country:
- U.S.
- Canada
- Latin America
- By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- By Country:
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- Europe
- By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- By Country:
- Germany
- U.K.
- France
- Italy
- Spain
- Russia
- Rest of Europe
- Asia Pacific
- By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- By Country:
- China
- India
- Japan
- Australia
- South Korea
- ASEAN
- Rest of Asia Pacific
- Middle East
- By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- By Country:
- GCC
- Israel
- Rest of Middle East
- Africa
- By Drug:
- Approved
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- IONIS-HTT Rx (RG6042)
- By Indication:
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Spinal Muscular Atrophy
- Huntington’s Disease
- By Distribution Channel:
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- By Country:
- South Africa
- Central Africa
- North Africa
- Company Profiles
- Alnylam Pharmaceuticals Inc.*
- Company Overview
- Product Portfolio
- Financial Performance
- Key Highlights
- Market Strategies
- Sarepta Therapeutics Inc.
- Biogen Inc.
- Ionis Pharmaceuticals Inc.
- Wave Life Sciences Ltd.
- Stroke Therapeutic Inc.
- Dynacure
- ProQR Therapeutics N.V.
- Q-STATE BIOSCIENCES, INC.
“*” marked represents similar segmentation in other categories in the respective section.
Table of Contents
- Research Objectives and Assumptions
- Research Objectives
- Assumptions
- Abbreviations
- Market Purview
- Report Description
- Market Definition and Scope
- Executive Summary
- Market Snippet, By Drug
- Market Snippet, By Indication
- Market Snippet, By Distribution Channel
- Market Snippet, By Region
- Coherent Opportunity Map (COM)
- Market Dynamics, Regulations, and Trends Analysis
- Market Dynamics
- Drivers
- Restraints
- Market Opportunities
- Impact Analysis
- Pipeline Analysis
- Reimbursement Scenario
- Epidemiology
- Pest Analysis
- Global CNS Specific Antisense Oligonucleotide Market, By Drug, 2018 – 2026, (US$ Mn)
- Introduction
- Market Share Analysis, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, 2017 – 2026
- Segment Trends
- Approved
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Patisiran
- Nusinersen
- Inotersen
- Pipeline
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- IONIS-HTT Rx (RG6042)
- Global CNS Specific Antisense Oligonucleotide Market, By Indication, 2018 – 2026, (US$ Mn)
- Introduction
- Market Share Analysis, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, 2017 – 2026
- Segment Trends
- Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Spinal Muscular Atrophy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Huntington’s Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Global CNS Specific Antisense Oligonucleotide Market, By Distribution Channel, 2018 – 2026, (US$ Mn)
- Introduction
- Market Share Analysis, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, 2017 – 2026
- Segment Trends
- Hospital Pharmacy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Retail Pharmacy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Online Pharmacy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016 – 2026, (US$ Mn)
- Global CNS Specific Antisense Oligonucleotide Market, By Region, 2018 – 2026, (US$ Mn)
- 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 Drug, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Indication, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
- U.S.
- Canada
- Latin America
- Market Size and Forecast, By Drug, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Indication, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- Europe
- Market Size and Forecast, By Drug, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Indication, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
- Germany
- U.K.
- France
- Italy
- Spain
- Russia
- Rest of Europe
- Asia Pacific
- Market Size and Forecast, By Drug, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Indication, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
- China
- India
- Japan
- Australia
- South Korea
- ASEAN
- Rest of APAC
- Middle East
- Market Size and Forecast, By Drug, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Indication, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
- GCC
- Israel
- Rest of Middle East
- Africa
- Market Size and Forecast, By Drug, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Indication, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026, (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026, (US$ Mn)
- South Africa
- Central Africa
- North Africa
- Competitive Landscape
- Heat Map Analysis
- Company Profiles
- Alnylam Pharmaceuticals Inc.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Sarepta Therapeutics Inc.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Biogen Inc.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Ionis Pharmaceuticals Inc.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Wave Life Sciences Ltd.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Stroke Therapeutic Inc.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Dynacure
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- ProQR Therapeutics N.V.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Q-STATE BIOSCIENCES, INC.
- Company Overview
- Drug Portfolio
- Financial Overview
- Key Highlights
- Market Strategies
- Analyst Views
- Section
- References
- Research Methodology
- About us and Sales Contact
*Browse 38 market data tables and 32 figures on "CNS Specific Antisense Oligonucleotide 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.
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.
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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