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

Table of Contents

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

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

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