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