OpportunityAnalyzer: Fragile X Syndrome - Opportunity Analysis and Forecasts to 2026

OpportunityAnalyzer: Fragile X Syndrome - Opportunity Analysis and Forecasts to 2026


In this report, GlobalData analyzes the nuances of the Fragile X Syndrome (FXS) treatment landscape and provides detailed insights into the market dynamics of this highly competitive space. The analysis also includes the evaluation of the commercial and clinical profiles of pipeline drugs, and their sales forecasts for the Fragile X Syndrome indication over the 2016-2026 period.

Although a relatively rare disease, Fragile X Syndrome has a distinct underlying genetic cause. Early intervention is key to the prognosis of the patient, yet there are currently no approved therapies specifically for Fragile X Syndrome. Instead, symptoms are managed on a case-by-case basis. In addition, a high rate of late-stage pipeline drugs have failed in clinical trials, meaning there are currently only four drugs with the potential to be launched during the forecast period. However, these drugs have a high potential for fast uptake in the market, as they would be the only approved therapies for Fragile X syndrome available and they each target their unique set of symptoms associated with the disease; the fast uptake of these pipeline drugs will be a principal driver of growth over the forecast period. However, lucrative opportunities remain for companies and products that improve upon one or more of unmet needs that will remain after the forecast period.


  • Overview of FXS, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
  • Annualized FXS therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in three patient segments (0-9 Year Olds, 10-19 Year Olds, 20+ Year Olds), forecast from 2016 to 2026.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the FXS therapeutics market.
  • Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for FXS therapy. The most promising candidates in Phase IIb and Phase III developments are profiled.
  • Analysis of the current and future market competition in the global FXS therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

  • Reasons to buy

    The report will enable you to -
    • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
    • Develop business strategies by understanding the trends shaping and driving the global FXS therapeutics market.
    • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global FXS therapeutics market in the future.
    • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
    • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
    • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Executive Summary
2.1 The FXS Market Will Experience Double-Digit Growth from 2016 to 2026
2.2 R&D Within the FXS Market
2.3 The First Therapies Approved Specifically for Fragile X Are on the Horizon
2.4 Market Opportunities for New Entrants
2.5 The Launch of Late-Stage Pipeline Drugs Will Be the Principal Driver of Sales Growth
2.6 What Do Physicians Think?
3 Introduction
3.1 Catalyst
3.2 Related Reports
4 Disease Overview
4.1 Etiology and Pathophysiology
4.1.1 Etiology
4.1.2 Pathophysiology
4.2 Classification or Staging Systems
4.3 Symptoms
4.4 Quality of Life
5 Epidemiology
5.1 Disease Background
5.2 Risk Factors and Comorbidities
5.3 Global and Historical Trends
5.4 Forecast Methodology
5.4.1 Sources
5.4.2 Forecast Assumptions and Methods
5.5 Epidemiological Forecast for FXS (2016-2026)
5.5.1 Diagnosed Prevalent Cases of FXS
5.5.2 Age-Specific Diagnosed Prevalent Cases of FXS
5.5.3 Sex-Specific Diagnosed Prevalent Cases of FXS
5.6 Discussion
5.6.1 Epidemiological Forecast Insight
5.6.2 Limitations of Analysis
5.6.3 Strengths of Analysis
6 Current Treatment Options
6.1 Overview
6.2 Diagnosis and Treatment
6.2.1 Diagnosis
6.2.2 Treatment Guidelines and Leading Prescribed Drugs
6.3 Clinical Practice
6.4 Selective Serotonin Reuptake Inhibitors
6.4.1 Overview
6.4.2 Efficacy and Safety
6.4.3 SWOT Analysis
6.5 Other Antidepressants
6.5.1 Overview
6.5.2 Efficacy and Safety
6.5.3 SWOT Analysis
6.6 Psychostimulants
6.6.1 Overview
6.6.2 Efficacy
6.6.3 Safety
6.6.4 SWOT Analysis
6.7 Atypical Antipsychotics
6.7.1 Overview
6.7.2 Efficacy
6.7.3 Safety
6.7.4 SWOT Analysis
6.8 Anticonvulsants
6.8.1 Overview
6.8.2 Efficacy
6.8.3 Safety
6.8.4 SWOT Analysis
6.9 Lithium
6.9.1 Overview
6.9.2 Efficacy
6.9.3 Safety
6.9.4 SWOT Analysis
6.10 Other Therapeutic Classes
7 Unmet Needs and Opportunity Assessment
7.1 Overview
7.2 Medication Specifically Approved for FXS Treatment
7.2.1 Unmet Need
7.2.2 Gap Analysis
7.2.3 Opportunity
7.3 Improved Efficacy and Safety Profile of Current Treatments
7.3.1 Unmet Need
7.3.2 Gap Analysis
7.3.3 Opportunity
7.4 Improved Clinical Trial Design
7.4.1 Unmet Need
7.4.2 Gap Analysis
7.4.3 Opportunity
7.5 Early Diagnosis Capability for Early Intervention and Improved Prognosis
7.5.1 Unmet Need
7.5.2 Gap Analysis
7.5.3 Opportunity
8 R&D Strategies
8.1 Overview
8.1.1 Modulating Inhibitory Neurotransmission
8.1.2 Modulating Excitatory Neurotransmission
8.1.3 Other Therapeutic Approaches
8.2 Clinical Trial Design
8.2.1 Lack of High-Quality Outcome Measures
8.2.2 Biomarkers for FXS
8.2.3 Consensus on Outcome Measures
9 Pipeline Assessment
9.1 Overview
9.2 Acamprosate Calcium
9.2.1 Overview
9.2.2 Efficacy
9.2.3 Safety
9.2.4 SWOT Analysis
9.3 Cannabidiol (ZYN002)
9.3.1 Overview
9.3.2 Efficacy
9.3.3 Safety
9.3.4 SWOT Analysis
9.4 Ganaxolone
9.4.1 Overview
9.4.2 Efficacy
9.4.3 Safety
9.4.4 SWOT Analysis
9.5 Trofinetide
9.5.1 Overview
9.5.2 Efficacy
9.5.3 Safety
9.5.4 SWOT Analysis
9.6 Innovative Early-Stage Approaches
9.6.1 Bryostatin-1
9.6.2 Gaboxadol
9.6.3 AMO-01
10 Pipeline Valuation Analysis
10.1 Clinical Benchmark of Key Pipeline Drugs
10.2 Commercial Benchmark of Key Pipeline Drugs
10.3 Competitive Assessment
10.4 Top-Line 10-Year Forecast
10.4.1 US
10.4.2 5EU
10.4.3 Japan
11 Appendix
11.1 Bibliography
11.2 Abbreviations
11.3 Methodology
11.4 Forecasting Methodology
11.4.1 Diagnosed Patients
11.4.2 Percent Drug-Treated Patients
11.4.3 Drugs Included in Each Therapeutic Class
11.4.4 Launch Dates
11.4.5 General Pricing Assumptions
11.4.6 Individual Drug Assumptions
11.4.7 Generic Erosion
11.4.8 Pricing of Pipeline Agents
11.5 Primary Research - KOLs Interviewed for This Report
11.6 Primary Research - Prescriber Survey
11.7 About the Authors
11.7.1 Analyst
11.7.2 Therapy Area Director
11.7.3 Epidemiologist
11.7.4 Managing Epidemiologist
11.7.5 Global Director of Therapy Analysis and Epidemiology
11.7.6 Global Head and EVP of Healthcare Operations and Strategy
11.8 About GlobalData
11.9 Contact Us
11.10 Disclaimer
1.1 List of Tables
Table 1: Fragile X Syndrome: Key Metrics in the 7MM
Table 2: Expansion Risk from Permutation to Full Mutation in Women
Table 3: Symptoms and Characteristics Associated with Fragile X Syndrome Description
Table 4: Comorbidities for FXS
Table 5: 7MM, Diagnosed Prevalent Cases of FXS, All Ages, Both Sexes, N, 2016-2026
Table 6: Treatment Guidelines for FXS
Table 7: Most Prescribed Drug Classes for FXS in the Global Markets, 2017
Table 8: Product Profile - SSRIs
Table 9: SSRIs SWOT Analysis, 2017
Table 10: Product Profile - Other Antidepressants
Table 11: Antidepressant SWOT Analysis, 2017
Table 12: Product Profile - Psychostimulants
Table 13: Psychostimulants SWOT Analysis, 2017
Table 14: Product Profile - Atypical Antipsychotics
Table 15: Atypical Antipsychotic SWOT Analysis, 2017
Table 16: Product Profile - Anticonvulsants
Table 17: Anticonvulsant SWOT Analysis, 2017
Table 18: Product Profile - Lithium
Table 19: Lithium SWOT Analysis, 2017
Table 20: Summary of Minor Therapeutic Classes, 2017
Table 21: Key Target Therapy Strategies Pursued by FXS Drug Developers
Table 22: Key Phase II/III Trials for the Promising Pipeline Agents that GlobalData Expects be Licensed for FXS in the 7MM During the Forecast Period
Table 23: Product Profile - Acamprosate Calcium
Table 24: Ongoing Phase III Study of Acamprosate Calcium in the US
Table 25: Acamprosate Calcium SWOT Analysis, 2017
Table 26: Product Profile - Cannabidiol
Table 27: Ongoing Phase I/II FAB-C Study of Cannabidiol in Australia
Table 28: Cannabidiol SWOT Analysis, 2017
Table 29: Product Profile - Ganaxolone
Table 30: Key Results from the Phase II Study of Ganaxolone in Belgium and the US
Table 31: Ganaxolone SWOT Analysis, 2017
Table 32: Product Profile - Trofinetide
Table 33: Trofinetide SWOT Analysis, 2017
Table 34: Innovative Early-Stage Approaches for FXS, 2017
Table 35: Clinical Benchmark of Key Pipeline Drugs - FXS
Table 36: Commercial Benchmark of Key Pipeline Drugs - FXS
Table 37: Key Events Impacting Sales for FXS, 2016-2026
Table 38: FXS Market - Global Drivers and Barriers, 2016-2026
Table 39: Key Historical and Projected Patent Expiry Dates for FXS
Table 40: Key Launch Dates for FXS in the 7MM
Table 41: High-Prescribing Physicians (non-KOLs) Surveyed, By Country
1.2 List of Figures
Figure 1: Global Sales Forecast by Country for Fragile X Syndrome in 2016 and 2026
Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against Methylphenidate
Figure 3: Genetic Location of FM1 Gene With Locus Xq27.3
Figure 4: Illustration of mGluR Theory of FXS
Figure 5: Typical Phenotypic Characteristics of FXS
Figure 6: 7MM, Sources Used, Diagnosed Prevalent Cases of FXS
Figure 7: 7MM, Age-Specific Diagnosed Prevalent Cases of FXS, Both Sexes, N, 2016
Figure 8: 7MM, Sex-Specific Diagnosed Prevalent Cases of FXS, All Ages, N, 2016
Figure 9: Unmet Need and Opportunity in Fragile X Syndrome, 2017
Figure 10: Overview of the Development Pipeline in FXS
Figure 11: Competitive Assessment of the FXS Pipeline Drugs Benchmarked Against Methylphenidate
Figure 12: Global (7MM) Sales Forecast by Country for FXS in 2016 and 2026
Figure 13: Global Sales Forecast by Class for FXS in 2016 and 2026
Figure 14: Sales Forecast by Class for FXS in the US in 2016 and 2026
Figure 15: Sales Forecast by Class for FXS in the 5EU in 2016 and 2026
Figure 16: Sales Forecast by Class for FXS in Japan in 2016 and 2026

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