UK Fragile-X Syndrome Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2603

  • Format: PPT, PDF

UK Fragile X Syndrome Therapeutics Market: Segmented by Diagnosis, Treatment, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

 

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SKU: 2603 Categories: ,

Report Overview

The UK Fragile X Syndrome Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Fragile X Syndrome (FXS), also termed Martin-Bell syndrome or marker X syndrome, is the most common cause of inherited mental retardation, intellectual disability, and autism and is the second most common cause of genetically associated mental deficiencies, after trisomy 21.

Fragile X syndrome is characterized by moderate intellectual disability in affected males and mild intellectual disability in affected females. Distinctive physical features are sometimes present in affected males including a large head, long face, prominent forehead and chin, protruding ears, loose joints, and large testes, but these features develop over time and may not be obvious until puberty. Behavioral abnormalities including autistic behaviors are common.

Fragile X syndrome is one of the most common forms of inherited learning disability; it may also cause associated physical conditions. Prevalence estimates from a UK screening study estimated an overall prevalence of 1 in 4,000 boys and 1 in 8000 girls. As the gene for the fragile X is located on the X chromosome the prevalence is less in females. Men are more often affected by this disorder than women. About 15% of men and 5% of women suffering from fragile X syndrome can experience seizures. The criterion standard diagnostic test for fragile X syndrome involves molecular genetic techniques that detect the FMR1 gene.

Market Growth Drivers

However, the presence of a strong pipeline that increases the focus of pharmaceuticals & biotechnology companies on conducting research programs for the effective treatment of syndrome also boosts up the market growth.

Moreover, rising cases of genetic mutation syndromes and increases in unhealthy lifestyles (such as smoking cigarettes, alcohol drinking, and others) by women during pregnancy also boost up the market growth.

High investment in research & development by leading industry participants is expected to open a new growth window for them. With strong research and development initiative, most of the companies are focusing on innovative and cost-effective drug development

Huge financial support to the researchers for developing novel interventions is boosting the market growth. The high demand for disease-specific novel treatment can also act as a market driver.

Market Restraints

However, the high cost of treatment and the lack of awareness for Fragile X Syndrome acts as a major restraint and as well as may hamper the market growth over the forecast period.

TABLE OF CONTENTS

1. UK Fragile X Syndrome Therapeutics Market Overview………………………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Presence of Strong Pipeline & High Investments in R&D Activities
B. Rising Cases of Genetic Mutation & Unhealthy Lifestyles
C. Huge Financial Support to the Researchers for Developing Novel Interventions
D. High Demand of Disease-Specific Novel Treatment
Market Restraints
A. Higher Cost of Treatment and Lower Treatment Rates
B. Limited Operating Revenue Opportunities by Many Market Players
3. Major Types of Fragile X Disorders…………………………………………………….
A. Fragile X Syndrome
B. Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS)
C. Fragile X-Associated Primary Ovarian Insufficiency (FXPOI)
4. Fragile X Syndrome Therapeutics Market Segmentation…………………..
A. By Diagnosis
I. Molecular Tests
II. Polymerase Chain Reaction (PCR)
III. Southern Blot Technique
IV. Molecular Genetic Testing
V. Others
B. By Treatment
I. Psychological Therapy
II. Pharmacological Therapy
III. Speech Therapy
IV. Sensory Integration Therapy
V. Occupational Therapy
VI. Behavioural Therapy
VII. Selective Serotonin Reuptake Inhibitors (SSRIs)
VIII. Antidepressants
IX. Atypical Antipsychotics
X. Alpha Agonists
XI. Others
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Fragile X Syndrome Major Drugs Market Share………………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Novartis Company overview, Product & Services, Strategies & Financials
B. Roche Company overview, Product & Services, Strategies & Financials
C. Ovid Company overview, Product & Services, Strategies & Financials
D. Zynerba Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………
A. New Trends and Development of Fragile X Syndrome Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

On the basis of diagnosis, the Fragile X syndrome treatment market can be classified into

  • Molecular Tests
  • Polymerase Chain Reaction (PCR)
  • Southern Blot
  • Molecular genetic testing
  • Others

There are molecular tests that allow the quantification of messenger RNA (mRNA) and FMRP protein, allowing a better understanding of the physiopathology of the disease. Polymerase chain reaction (PCR) and Southern blot are the routine tests for the DNA diagnosis of FXS, which allow determining the number of CGG repeats and the methylation status of the FMR1 gene. The PCR through the use of specific primers for the FMR1 gene allows the amplification of the region that contains the CGG repeat and, can identify patients with an expanded FMR1 allele particularly in the premutation but also in the full mutation range. Molecular genetic testing for mutations in the specific genes known to cause FXS confirms the diagnosis of the disease.

By Treatment:

Based on the treatment, the Fragile X syndrome treatment market can be bifurcated into

  • Psychological Therapy
  • Pharmacological Therapy
  • Speech Therapy
  • Sensory Integration Therapy
  • Occupational Therapy
  • Behavioural Therapy
  • Selective Serotonin Reuptake Inhibitors (SSRIs)
  • Antidepressants
  • Atypical Antipsychotics
  • Alpha Agonists
  • Others

Current approaches to therapy for FXS are all symptom-based, and few controlled trials have been performed to determine their effectiveness. Psychopharmacologic intervention should be combined with other supportive strategies, including speech therapy, sensory integration, occupational therapy, individualized educational plans, and tailored behavioral interventions to maximize functioning. The treatment regimen of FXS includes the use of stimulants, selective serotonin reuptake inhibitors (SSRIs), atypical antipsychotics, and alpha agonists.

By Distribution Channels:

In terms of distribution channel, the Fragile X Syndrome (FXS) treatment market can be classified into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

In terms of value, the hospital pharmacies accounted for the leading market share in 2020, which is attributable to the fact that the majority of the medications for Fragile X syndrome treatment are dispensed following by the prescription there and can so only be done by trained medical professionals. Hence, the hospital pharmacies segment is anticipated to enhance the market share over the forecast period.

Top Major Players

The UK Fragile X Syndrome treatment market is an attractive market and the prominent players are focusing on organic growth strategies such as product launches, product approvals, and others such as patents and events. Additionally, the inorganic growth strategies activities witnessed in the market are acquisitions, and partnerships & collaborations. These activities have paved the way for the expansion of the business and the customer base of market players. Major players operating in the market include Novartis Pharma UK, F. Hoffmann-La Roche Ltd, Ovid Therapeutics, and Zynerba Pharmaceuticals Limited among others.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

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Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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