Summary
Huntington’s Disease (HD) is a rare, autosomal-dominant, neurodegenerative disorder, characterized by impaired motor control, cognitive dysfunction, behavioral changes, and mood disorders. Approved treatments for HD across the 7MM are extremely limited, consisting of only two symptomatic treatments, tetrabenazine and tiapride, which are both approved for the treatment of HD-associated chorea. Other treatment options contributing to the HD market are off-label symptomatic drugs, many of which are widely genericized throughout the 7MM. Four late-stage pipeline drugs, two symptomatic treatments (SD-809 and pridopidine) and two potential DMDs (RP103 and PBT2) are expected to launch in the US and 5EU markets during the forecast period, and will be a source of significant growth in the HD market. It is anticipated that SD-809 and pridopidine will also launch in Japan within the forecast period.
Highlights
Key Questions Answered
- The HD market has been dominated by tetrabenazine and tiapride (approved in Germany, Italy, and Spain; off-label in France and Japan) for many years, with little approved therapeutic options available in the 7MM. New classes of drugs are anticipated to reach the market within the forecast period. How will these change the market overall during the forecast period? Will tetrabenazine maintain market dominance in 2024?
- The current late stage HD pipeline is varied, with first-in-class drugs in development with innovative modes of action. Which of these will have the biggest impact on the market? What strategies are developers undertaking to penetrate this chronically underserved market? Will these fulfil any unmet needs?
Key Findings
- The main driver of growth in the HD market will be the introduction of premium-priced disease-modifying drugs and improved symptomatic therapies over the forecast period. The launch of these treatments will act as a stimulant for growth in this chronically underserved market.
- Significant barriers to growth in the HD market will be the high cost of novel treatments and potential issues surrounding reimbursement, which will limit the use of more expensive drugs. Patients who have an acceptable level of symptomatic control with currently marketed drugs could result in limited treatment costs for HD.
- The arrival of new therapies across the 7MM will increase treatment rates and provide alternative options for many patients.
- The largest unmet needs in the HD market are the development of efficacious disease-modifying drugs and symptomatic treatments. In addition, the identification of a robust HD biomarker for monitoring disease progression, as well as determining the effects of therapeutic interventions, is a significant unmet need in this field.
Scope
- Overview of HD, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
- Annualized HD market revenue, annual cost of therapy and treatment usage pattern data from 2014 and forecast for ten years to 2024.
- Key topics covered include market characterization, unmet needs, R&D and clinical trials assessment, late stage clinical trial analysis and implications for the HD therapeutics market.
- Pipeline analysis: focus on the six late-stage pipeline HD drugs discussing emerging trends as well as overview of earlier phase drugs.
- Analysis of the current and future market competition in the global HD 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. Additionally a list of acquisition targets included in the pipeline product company list.
- Develop business strategies by understanding the trends shaping and driving the global HD therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global HD therapeutics market in 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.
- Track drug sales in the global HD therapeutics market from 2014-2024.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
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1.1 List of Tables
1.2 List of Figures
2 Introduction
2.1 Catalyst
2.2 Related Reports
3 Disease Overview
3.1 Etiology and Pathophysiology
3.1.1 Etiology
3.1.2 Pathophysiology
3.2 Quality of Life
3.3 Symptoms
3.4 Diagnosis and Disease Monitoring
4 Epidemiology
4.1 Disease Background
4.2 Risk Factors and Complications
4.3 Global Trends
4.3.1 Diagnosed Prevalence of HD-7MM
4.4 Forecast Methodology
4.4.1 Sources Used
4.4.2 Sources Not Used
4.4.3 Forecast Assumptions and Methods
4.5 Epidemiological Forecast of HD (2014-2024)
4.5.1 Diagnosed Prevalent Cases
4.5.2 Diagnosed Prevalent Cases of HD by Age at Onset
4.5.3 Diagnosed Prevalent Cases of Adults with Juvenile-Onset HD that Progressed to Adulthood, Ages =20 Years
4.6 Discussion
4.6.1 Epidemiological Forecast Insight
4.6.2 Limitations of the Analysis
4.6.3 Strengths of the Analysis
5 Current Treatment Options
5.1 Overview
5.1.1 Antipsychotic Drugs
5.1.2 Antidepressant Drugs
5.1.3 Benzodiazepines
5.1.4 Anticonvulsant Drugs
5.1.5 Antiparkinson Drugs
5.1.6 Botulinum Toxin Type A
5.2 Product Profiles-Major Brands
5.2.1 Xenazine (Tetrabenazine)
5.2.2 Tiapride
6 Unmet Needs Assessment and Opportunity Analysis
6.1 Overview
6.2 Disease-Modifying Drugs
6.2.1 Unmet Need
6.2.2 Gap Analysis
6.2.3 Opportunity
6.3 Efficacious Cognitive Enhancers
6.3.1 Unmet Need
6.3.2 Gap Analysis
6.3.3 Opportunity
6.4 Improved Symptomatic Treatments
6.4.1 Unmet Need
6.4.2 Gap Analysis
6.4.3 Opportunity
6.5 Identification of Disease Biomarker
6.5.1 Unmet Need
6.5.2 Gap Analysis
6.5.3 Opportunity
6.6 Increased Knowledge of HD Pathogenesis
6.6.1 Unmet Need
6.6.2 Gap Analysis
6.6.3 Opportunity
6.7 More Comprehensive Treatment Guidelines
6.7.1 Unmet Need
6.7.2 Gap Analysis
6.7.3 Opportunity
6.8 Novel Formulations
6.8.1 Unmet Need
6.8.2 Gap Analysis
6.8.3 Opportunity
7 R&D Strategies
7.1 Overview
7.1.1 Disease-Modifying Drugs
7.1.2 Clinical Measures and Biomarker Identification
7.2 Clinical Trial Design
7.2.1 Efficacy Endpoints
7.2.2 Patient Inclusion
7.2.3 Trial Locations
7.2.4 Type of Clinical Trial
8 Pipeline Assessment
8.1 Overview
8.1.1 SD-809 (Deutetrabenazine)
8.1.2 Pridopidine Hydrochloride
8.1.3 RP103 (Cysteamine Bitartrate)
8.1.4 PF-02545920
8.1.5 Laquinimod
8.1.6 PBT2
8.2 Innovative Early-Stage Approaches
8.2.1 VX-15
8.2.2 IONIS-HTTRx
9 Pipeline Valuation Analysis
9.1 Clinical Benchmark of Key Pipeline Drugs
9.2 Commercial Benchmark of Key Pipeline Drugs
9.3 Competitive Assessment
9.4 Top-Line 10-Year Forecast
9.4.1 US
9.4.2 5EU
9.4.3 Japan
10 Appendix
10.1 Bibliography
10.2 Abbreviations
10.3 Methodology
10.4 Forecasting Methodology
10.4.1 Diagnosed HD Patients
10.4.2 Percent Drug-Treated Patients
10.4.3 Drugs Included in Each Therapeutic Class
10.4.4 Launch and Patent Expiry Dates
10.4.5 General Pricing Assumptions
10.4.6 Individual Drug Assumptions
10.4.7 Generic Erosion
10.4.8 Pricing of Pipeline Agents
10.5 Primary Research
10.5.1 Physicians and Specialists Included in this Study
10.5.2 Online Survey of High-Prescribing Physicians
10.6 About the Authors
10.6.1 Author
10.6.2 Reviewer
10.6.3 Therapy Area Director
10.6.4 Epidemiologist
10.6.5 Global Director of Therapy Analysis and Epidemiology
10.6.6 Global Head of Healthcare
10.7
10.8 Disclaimer
1.1 List of Tables
Table 1: Common Symptoms of HD
Table 2: The UHDRS Total Functional Capacity Scale
Table 3: The Shoulson-Fahn Huntington’s Disease Severity Scale
Table 4: Risk Factors and Complications for HD
Table 5: 7MM Estimates for the Diagnosed Prevalence of HD
Table 6: 7MM, Sources of Epidemiological Data Used for the Forecast of HD Diagnosed Prevalent Cases
Table 7: 7MM, Sources of Epidemiological Data Used for the Forecast of JHD Diagnosed Prevalent Cases
Table 8: 7MM, Sources of Epidemiological Data Used for the Forecast of HD Diagnosed Prevalent Cases by Age at Onset
Table 9: 7MM, Diagnosed Prevalent Cases of HD, All Ages, Both Sexes, N, 2014-2024
Table 10: 7MM, Diagnosed Prevalent Cases of HD by Age Group, Both Sexes, N (%), 2014
Table 11: 7MM, Sex-Specific Diagnosed Prevalent Cases of HD, All Ages, N (%), 2014
Table 12: 7MM, Diagnosed Prevalent Cases of JHD, Age <20 Years, Both Sexes, N, 2014-2024
Table 13: 7MM, Age-Specific Diagnosed Prevalent Cases of JHD, Both Sexes, N (%), 2014
Table 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of JHD, Age <20 Years, N (%), 2014
Table 15: 7MM, Diagnosed Prevalent Cases of Adults with Juvenile-Onset HD that Progressed to Adulthood, Age =20 Years, Both Sexes, N, 2014-2024
Table 16: Leading Treatments for HD, 2016
Table 17: Antipsychotics SWOT Analysis, 2016
Table 18: Antidepressants SWOT Analysis, 2016
Table 19: Benzodiazepines SWOT Analysis, 2016
Table 20: Anticonvulsants SWOT Analysis, 2016
Table 21: Antiparkinson Drugs SWOT Analysis, 2016
Table 22: Botulinum Toxin Type A SWOT Analysis, 2016
Table 23: Product Profile-Xenazine
Table 24: Xenazine SWOT Analysis, 2016
Table 25: Product Profile-Tiapride
Table 26: Tiapride SWOT Analysis, 2016
Table 27: Overall Unmet Needs-Current Level of Attainment
Table 28: HD-Late-Stage Pipeline, 2016
Table 29: Product Profile-SD-809
Table 30: Summary of Treatment Effects of SD-809 on the Motor Endpoints in the FIRST-HD Clinical Trial
Table 31: Summary of Treatment Effects of SD-809 on the Secondary Endpoints in the FIRST-HD Clinical Trial
Table 32: SD-809 SWOT Analysis, 2016
Table 33: Product Profile-Pridopidine
Table 34: Summary of Adverse Events in the MermaiHD Clinical Trial of Pridopidine
Table 35: Summary of the Most Common Adverse Events in the HART Clinical Trial of Pridopidine
Table 36: Pridopidine SWOT Analysis, 2016
Table 37: Product Profile-RP103
Table 38: RP103 SWOT Analysis, 2016
Table 39: Product Profile-PF-02545920
Table 40: PF-02545920 SWOT Analysis, 2016
Table 41: Product Profile-Laquinimod
Table 42: Laquinimod SWOT Analysis, 2016
Table 43: Product Profile-PBT2
Table 44: Summary of the Adverse Events in the REACH2HD Clinical Trial of PBT2
Table 45: PBT2 SWOT Analysis, 2016
Table 46: Early-Stage Pipeline Products in HD
Table 47: Clinical Benchmark of Key Pipeline Drugs-HD
Table 48: Commercial Benchmark of Key Pipeline Drugs-HD
Table 49: Top-Line Sales Forecast ($m) for HD, 2014-2024
Table 50: Key Events Impacting Sales for HD, 2014-2024
Table 51: HD Market-Drivers and Barriers, 2014-2024
Table 52: Key Launch Dates
Table 53: Key Patent Expiries
Table 54: High-Prescribing Physicians (Non-KOLs) Surveyed, By Country
1.2 List of Figures
Figure 1: Cortico-basal Ganglia Network Indicating the Direct and Indirect Pathways
Figure 2: 7MM, Diagnosed Prevalent Cases of HD, All Ages, Both Sexes, N, 2014-2024
Figure 3: 7MM, Diagnosed Prevalent Cases of HD by Age Group, Both Sexes, N, 2014
Figure 4: 7MM, Diagnosed Prevalent Cases of HD by Sex, All Ages, N, 2014
Figure 5: 7MM, Age-Standardized Diagnosed Prevalence of HD (%) by Sex, All Ages, 2014
Figure 6: 7MM, Diagnosed Prevalent Cases of JHD, Age <20 Years, Both Sexes, N, 2014-2024
Figure 7: 7MM, Diagnosed Prevalent Cases of JHD by Age Group, Both Sexes, N
Figure 8: 7MM, Diagnosed Prevalent Cases of JHD by Sex, Age <20 Years, N, 2014
Figure 9: 7MM, Diagnosed Prevalent Cases of HD by Age at Onset, Both Sexes, N, 2014
Figure 10: 7MM, Diagnosed Prevalent Cases of Adults with Juvenile-Onset HD that Progressed to Adulthood, Age =20 Years, Both Sexes, N, 2014
Figure 11: Competitive Assessment of Marketed and Late-Stage Pipeline HD Drugs, 2014-2024
Figure 12: Sales for HD by Region, 2014-2024
1.1 List of Tables
Table 1: Common Symptoms of HD
Table 2: The UHDRS Total Functional Capacity Scale
Table 3: The Shoulson-Fahn Huntington’s Disease Severity Scale
Table 4: Risk Factors and Complications for HD
Table 5: 7MM Estimates for the Diagnosed Prevalence of HD
Table 6: 7MM, Sources of Epidemiological Data Used for the Forecast of HD Diagnosed Prevalent Cases
Table 7: 7MM, Sources of Epidemiological Data Used for the Forecast of JHD Diagnosed Prevalent Cases
Table 8: 7MM, Sources of Epidemiological Data Used for the Forecast of HD Diagnosed Prevalent Cases by Age at Onset
Table 9: 7MM, Diagnosed Prevalent Cases of HD, All Ages, Both Sexes, N, 2014-2024
Table 10: 7MM, Diagnosed Prevalent Cases of HD by Age Group, Both Sexes, N (%), 2014
Table 11: 7MM, Sex-Specific Diagnosed Prevalent Cases of HD, All Ages, N (%), 2014
Table 12: 7MM, Diagnosed Prevalent Cases of JHD, Age <20 Years, Both Sexes, N, 2014-2024
Table 13: 7MM, Age-Specific Diagnosed Prevalent Cases of JHD, Both Sexes, N (%), 2014
Table 14: 7MM, Sex-Specific Diagnosed Prevalent Cases of JHD, Age <20 Years, N (%), 2014
Table 15: 7MM, Diagnosed Prevalent Cases of Adults with Juvenile-Onset HD that Progressed to Adulthood, Age =20 Years, Both Sexes, N, 2014-2024
Table 16: Leading Treatments for HD, 2016
Table 17: Antipsychotics SWOT Analysis, 2016
Table 18: Antidepressants SWOT Analysis, 2016
Table 19: Benzodiazepines SWOT Analysis, 2016
Table 20: Anticonvulsants SWOT Analysis, 2016
Table 21: Antiparkinson Drugs SWOT Analysis, 2016
Table 22: Botulinum Toxin Type A SWOT Analysis, 2016
Table 23: Product Profile-Xenazine
Table 24: Xenazine SWOT Analysis, 2016
Table 25: Product Profile-Tiapride
Table 26: Tiapride SWOT Analysis, 2016
Table 27: Overall Unmet Needs-Current Level of Attainment
Table 28: HD-Late-Stage Pipeline, 2016
Table 29: Product Profile-SD-809
Table 30: Summary of Treatment Effects of SD-809 on the Motor Endpoints in the FIRST-HD Clinical Trial
Table 31: Summary of Treatment Effects of SD-809 on the Secondary Endpoints in the FIRST-HD Clinical Trial
Table 32: SD-809 SWOT Analysis, 2016
Table 33: Product Profile-Pridopidine
Table 34: Summary of Adverse Events in the MermaiHD Clinical Trial of Pridopidine
Table 35: Summary of the Most Common Adverse Events in the HART Clinical Trial of Pridopidine
Table 36: Pridopidine SWOT Analysis, 2016
Table 37: Product Profile-RP103
Table 38: RP103 SWOT Analysis, 2016
Table 39: Product Profile-PF-02545920
Table 40: PF-02545920 SWOT Analysis, 2016
Table 41: Product Profile-Laquinimod
Table 42: Laquinimod SWOT Analysis, 2016
Table 43: Product Profile-PBT2
Table 44: Summary of the Adverse Events in the REACH2HD Clinical Trial of PBT2
Table 45: PBT2 SWOT Analysis, 2016
Table 46: Early-Stage Pipeline Products in HD
Table 47: Clinical Benchmark of Key Pipeline Drugs-HD
Table 48: Commercial Benchmark of Key Pipeline Drugs-HD
Table 49: Top-Line Sales Forecast ($m) for HD, 2014-2024
Table 50: Key Events Impacting Sales for HD, 2014-2024
Table 51: HD Market-Drivers and Barriers, 2014-2024
Table 52: Key Launch Dates
Table 53: Key Patent Expiries
Table 54: High-Prescribing Physicians (Non-KOLs) Surveyed, By Country
