Tuesday, 02 January 2024 12:17 GMT

Huntington's Disease Market Insight, Epidemiology, And Market Forecast - 2036 Now Available - See Which Pipeline Candidates Are Racing To Become The First Disease-Modifying Therapy


(MENAFN- GlobeNewsWire - Nasdaq) 198-Page Report Tracks SKY-0515, Votoplam (PTC518), and the Gene-Silencing Pipeline as the $320M Market Shifts from Symptom Management Toward a Cure

Dublin, Sept. 08, 2026 (GLOBE NEWSWIRE) -- The "Huntington's Disease - Market Insight, Epidemiology, and Market Forecast - 2036" has been added to ResearchAndMarkets.com's offering.

The Huntington's disease market across the seven major markets (7MM)-the United States, Germany, France, Italy, Spain, the United Kingdom, and Japan-was valued at approximately USD 320 million in 2025. The market is forecast to grow at a compound annual growth rate (CAGR) of 14% from 2026 to 2036, supported by increasing diagnosis, greater research investment, and the development of gene-targeting and RNA-based therapies.

The "Huntington's Disease - Market Insights, Epidemiology and Market Forecast - 2036" report provides a comprehensive assessment of historical and projected epidemiology, current treatment practices, emerging therapies, market access, drug uptake, competitive dynamics, and commercial opportunities across the 7MM. The analysis covers the 2022-2036 period and evaluates the factors expected to reshape the Huntington's disease treatment landscape.

Huntington's Disease Market Highlights

  • The 7MM Huntington's disease market reached approximately USD 320 million in 2025.
  • The market is expected to expand at a 14% CAGR between 2026 and 2036.
  • AUSTEDO generated an estimated USD 167 million in 2025, representing the largest therapy-level revenue contribution.
  • Approved therapies remain focused primarily on chorea and other manifestations rather than disease modification.
  • Pipeline candidates including SKY-0515 and votoplam (PTC518) are advancing the shift toward huntingtin-lowering and RNA-targeted treatment strategies.
  • Genetic testing, biomarker development, patient advocacy, and clinical trial activity are expected to support market expansion.

Current Treatment Landscape and Unmet Medical Need

Huntington's disease treatment currently centers on symptom management and multidisciplinary supportive care. AUSTEDO (deutetrabenazine) from Teva Pharmaceuticals, INGREZZA (valbenazine) from Neurocrine Biosciences, and generic tetrabenazine are used to manage chorea. Antidepressants, antipsychotics, and mood stabilizers may be incorporated to address psychiatric and behavioral manifestations, while physical, occupational, and speech therapy support patient function.

The absence of an approved disease-modifying therapy remains the most significant unmet need. Additional clinical gaps include limited options for cognitive decline, inconsistent control of psychiatric symptoms, variability in disease progression, safety and tolerability concerns with long-term treatment, and challenges involving diagnosis and predictive testing. Drug development is also affected by central nervous system delivery barriers, limited clinical trial populations, regulatory uncertainty, and the need for validated biomarkers and standardized endpoints.

Huntington's Disease Epidemiology

The United States recorded approximately 44,000 prevalent Huntington's disease cases in 2025, while Germany, France, Italy, Spain, and the United Kingdom collectively accounted for approximately 37,000 cases. Prevalence is projected to increase through 2036. Individuals aged 60 years and older represented approximately 50% of total cases across the 7MM. In Japan, an estimated 80 Stage IV cases and 20 Stage V cases were identified in 2025.

Improved access to predictive genetic testing and advances in patient identification may expand the diagnosed population and facilitate earlier clinical intervention. Better characterization of disease progression and biomarker profiles is also expected to strengthen clinical trial design and support precision medicine strategies.

Approved Huntington's Disease Therapies

AUSTEDO is a VMAT2 inhibitor indicated for chorea associated with Huntington's disease in adults. In May 2024, the US Food and Drug Administration approved additional AUSTEDO XR tablet strengths, enabling a one-pill, once-daily option for eligible adults with Huntington's disease chorea or tardive dyskinesia. The estimated annual treatment cost is approximately USD 60,000, although patient assistance and reimbursement programs may reduce out-of-pocket expenses for qualifying patients.

INGREZZA is a selective VMAT2 inhibitor approved by the FDA in August 2023 for the treatment of chorea associated with Huntington's disease in adults. The approval was supported by the Phase III KINECT-HD study and the KINECT-HD2 open-label extension trial conducted with the Huntington Study Group.

Emerging Huntington's Disease Pipeline

SKY-0515, developed by Skyhawk Therapeutics, is an investigational oral RNA-modulating small molecule designed to reduce HTT and PMS1 proteins. The Phase II/III FALCON-HD program has expanded beyond Australia and New Zealand, with more than 90 patients reported as treated. In September 2025, interim Phase I findings showed dose-dependent reductions in blood mutant huntingtin protein, including a 62% reduction at the 9 mg dose by Day 84. In January 2026, Skyhawk reported positive results from a nine-month interim Phase I analysis.

Votoplam, also known as PTC518, is an investigational oral HTT mRNA splice modulator. In March 2025, the Phase II PIVOT-HD study met its primary endpoint by demonstrating a statistically significant reduction in blood HTT protein at Week 12, alongside favorable safety and tolerability findings. Twelve-month data from Stage 2 patients also supported dose-dependent HTT lowering and trends across clinical measures. Novartis entered a global license and collaboration agreement with PTC Therapeutics for the program in December 2024.

Other companies contributing to the Huntington's disease competitive landscape include Alnylam Pharmaceuticals, Roche, uniQure Biopharma, Teva Pharmaceuticals, Neurocrine Biosciences, Novartis, and Skyhawk Therapeutics. Pipeline development increasingly emphasizes antisense oligonucleotides, RNA interference, splice modulation, gene therapy, and other approaches intended to reduce mutant huntingtin expression or slow disease progression.

Huntington's Disease Market Outlook

The market is expected to transition gradually from symptomatic management toward mechanism-based therapies. Successful clinical development and commercialization of disease-modifying candidates could substantially increase treatment uptake, market revenue, and the addressable patient population. Oral therapies may offer additional adoption advantages, although efficacy, safety, durability, reimbursement, and patient selection will remain critical commercial factors.

The report evaluates therapy-level market share, peak patient share, anticipated drug uptake, pricing trends, reimbursement conditions, patient access, competitive positioning, and country-specific opportunities. It also incorporates epidemiology-based forecasting, SWOT analysis, conjoint analysis, physician perspectives, and assessments of treatment safety, efficacy, administration, market entry, and probability of success.

Report Coverage

  • Huntington's disease epidemiology and patient population forecasts across the 7MM
  • Historical and forecast market size from 2022 to 2036
  • Approved therapy profiles and Phase I-III pipeline analysis
  • Drug pricing, reimbursement, market access, and analogue assessment
  • Therapy uptake, peak patient share, and sales forecasts
  • Competitive intelligence and leading company analysis
  • Unmet medical needs, treatment practices, and clinical development trends
  • Expert insights, SWOT analysis, and conjoint analysis

The report is designed to support pharmaceutical companies, biotechnology developers, investors, healthcare providers, and market access teams seeking actionable insights into the Huntington's disease market. Its findings can inform clinical development, portfolio prioritization, commercialization planning, pricing strategy, competitive positioning, and geographic expansion through 2036.

Key Topics Covered:
1. Key Insights
2. Report Introduction
3. Executive Summary
4. Key Events
4.1. Upcoming Key Catalysts
4.2. Key Conferences and Meetings
4.3. Key Transactions and Collaborations
4.4. News Flow
5. Epidemiology and Market Forecast Methodology
6. Huntington's Disease Market Overview at a Glance
6.1. Emerging Landscape Analysis in 7MM (by Phase)
6.3. Market Size of Huntington's Disease by Line of Therapies in 7MM (2025)
6.4. Market Size of Huntington's Disease by Line of Therapies in 7MM (2036)
7. Disease Background and Overview of Huntington's Disease
7.1. Introduction
7.2. Causes of Huntington's Disease
7.3. Risk Factors
7.4. Patient-related Risk Factors
7.5. Signs and Symptoms
7.6. Pathogenesis
7.7. Pathophysiology
7.8. Diagnosis
7.9. Stages
8. Treatment and Management of Huntington's Disease
8.1. Adjunctive Treatment and Supportive Care
9. Patient Journey of Huntington's Disease
10. Epidemiology and Patient Population of Huntington's Disease
10.1. Key Findings
10.2. Assumptions and Rationale
10.2.1. 7MM
10.3. Total Prevalent Cases of Huntington's Disease
10.4. Total Diagnosed Prevalent Cases of Huntington's Disease
10.5. United States
10.5.1. Total Prevalent Cases of Huntington's Disease in the United States
10.5.2. Total Diagnosed Prevalent Cases of Huntington's Disease in the United States
10.5.3. Age-specific Cases of Huntington's Disease in the United States
10.5.4. Stage-specific Cases of Huntington's Disease in the United States
10.5.5. Total Treated Cases of Huntington's Disease in the United States
10.6. EU4 and the UK
10.6.1. Total Prevalent Cases of Huntington's Disease in EU4 and the UK
10.6.2. Total Diagnosed Prevalent Cases of Huntington's Disease in EU4 and the UK
10.6.3. Age-specific Cases of Huntington's Disease in EU4 and the UK
10.6.4. Stage-specific Cases of Huntington's Disease in EU4 and the UK
10.6.5. Total Treated Cases of Huntington's Disease in EU4 and the UK
10.7. Japan
10.7.1. Total Prevalent Cases of Huntington's Disease in Japan
10.7.2. Total Diagnosed Prevalent Cases of Huntington's Disease in Japan
10.7.3. Age-specific Cases of Huntington's Disease in Japan
10.7.4. Stage-specific Cases of Huntington's Disease in Japan
10.7.5. Total Treated Cases of Huntington's Disease in Japan
11. Marketed Drugs
11.1. Marketed Competitive Landscape of Huntington's Disease
11.2. Deutetrabenazine (AUSTEDO): Teva Pharmaceuticals
11.2.1. Product Description
11.2.2. Other Developmental Activities
11.2.3. Clinical Development
11.2.4. Clinical Trials Information
11.2.5. Safety and Efficacy
11.2.6. Analyst's View
11.3. INGREZZA (valbenazine/NBI-98854): Neurocrine Biosciences
11.3.1. Product Description
11.3.2. Other Developmental Activities
11.3.3. Clinical Development
11.3.4. Clinical Trials Information
11.3.5. Safety and Efficacy
11.3.6. Analyst's View
List to be continued in the report..
12. Emerging Drugs
12.1. Emerging Competitive Landscape of Huntington's Disease
12.2. SKY-0515: Skyhawk Therapeutics
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.3.1. Clinical Trial Information
12.2.4. Safety and Efficacy
12.2.5. Analyst's View
12.3. Votoplam (PTC518): Novartis Pharmaceuticals
12.3.1. Product Description
12.3.2. Other Developmental Activities
12.3.3. Clinical Development
12.3.3.1. Clinical Trial Information
12.3.4. Safety and Efficacy
12.3.5. Analyst's View
List to be continued in the report..
13. Huntington's Disease: 7MM Market Analysis
13.1. Key Findings
13.2. Market Outlook of Huntington's Disease
13.2.1. Market Dynamics: 7MM
13.3. Key Market Forecast Assumptions
13.3.1. Cost Assumptions and Rebate
13.3.2. Pricing Trends
13.3.3. Analogue Assessment
13.3.4. Launch Year and Therapy Uptake
13.4. Total Market Size of Huntington's Disease by Country in the 7MM
13.5. The United States
13.5.1. Total Market Size of Huntington's Disease in the United States
13.5.2. Total Market Size of Huntington's Disease by Therapies in the United States
13.6. EU4 and the UK
13.6.1. Total Market Size of Huntington's Disease in EU4 and the UK
13.6.2. Total Market Size of Huntington's Disease by Therapies in EU4 and the UK
13.7. Japan
13.7.1. Total Market Size of Huntington's Disease in Japan
13.7.2. Total Market Size of Huntington's Disease by Therapies in Japan
14. Unmet Needs of Huntington's Disease
15. SWOT Analysis of Huntington's Disease
16. KOL Views of Huntington's Disease
17. Market Access and Reimbursement of Huntington's Disease
17.1. The United States
17.2. In EU4 and the UK
17.2.1. Germany
17.2.2. France
17.2.3. Italy
17.2.4. Spain
17.2.5. United Kingdom
17.3. Japan
17.4. Summary and Comparison of Market Access and Pricing Policy Developments in 2025
17.5. Market Access and Reimbursement of Huntington's Disease
18. Appendix
18.1. Bibliography
18.2. Report Methodology
19. Analyst's Capabilities
20. Disclaimer
21. About the Publisher
List of Tables
Table 1: Summary of Huntington's disease: Market, Epidemiology, and Key Events (2022-2036)
Table 2: Genetic Testing for Huntington's Disease
Table 3: AAN Summary of Evidence-based Guideline for Clinicians
Table 4: Levels of evidence
Table 5: Grades of Recommendations
Table 6: Total prevalent cases of Huntington's Disease in the 7MM (2022-2036)
Table 7: Total Diagnosed Prevalent cases of Huntington's disease in the 7MM (2022-2036)
Table 8: Total Chorea associated cases of Huntington's disease in the 7MM (2022-2036)
Table 9: Total Diagnosed Prevalent cases of Huntington's disease by age group in the 7MM (2022-2036)
Table 10: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in the 7MM (2022-2036)
Table 11: Total Treated cases of Huntington's Disease in the 7MM (2022-2036)
Table 12: Total prevalent cases of Huntington's Disease in the US (2022-2036)
Table 13: Total Diagnosed Prevalent cases of Huntington's disease in the US (2022-2036)
Table 14: Total Chorea associated cases of Huntington's disease in the US (2022-2036)
Table 15: Total Diagnosed Prevalent cases of Huntington's disease by age group in the US (2022-2036)
Table 16: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in the US (2022-2036)
Table 17: Total Treated cases of Huntington's Disease in the US (2022-2036)
Table 18: Total prevalent cases of Huntington's Disease in the EU5 (2022-2036)
Table 19: Total Diagnosed Prevalent cases of Huntington's disease in the EU5 (2022-2036)
Table 20: Total Chorea associated cases of Huntington's disease in the EU5 (2022-2036)
Table 21: Total Diagnosed Prevalent cases of Huntington's disease by age group in the EU5 (2022-2036)
Table 22: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in the EU5 (2022-2036)
Table 23: Total Treated cases of Huntington's Disease in the EU5 (2022-2036)
Table 24: Total prevalent cases of Huntington's Disease in Japan (2022-2036)
Table 25: Total Diagnosed Prevalent cases of Huntington's disease in Japan (2022-2036)
Table 26: Total Chorea associated cases of Huntington's disease in Japan (2022-2036)
Table 27: Total Diagnosed Prevalent cases of Huntington's disease by age group in Japan (2022-2036)
Table 28: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in Japan (2022-2036)
Table 29: Total Treated cases of Huntington's Disease in Japan (2022-2036)
Table 30: Austedo (Deutetrabenazine/SD-809), Clinical Trial Description, 2022
Table 31: Pridopidine (ACR-16; Huntexil), Clinical Trial Description, 2022
Table 32: Ingrezza (valbenzine), Clinical Trial Description, 2022
Table 33: ANX005, Clinical Trial Description, 2022
Table 34: SOM3355/Bevantolol, Clinical Trial Description, 2022
Table 35: VX15/2503(pepinemab), Clinical Trial Description, 2022
Table 36: SAGE-718, Clinical Trial Description, 2022
Table 37: Branaplam (LMI070), Clinical Trial Description, 2022
Table 38: AMT-130, Clinical Trial Description, 2022
Table 39: WVE-003, Clinical Trial Description, 2022
Table 40: 7MM Market Size of Huntington's disease in USD Million (2022-2036)
Table 41: 7MM Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
Table 42: United States Market Size of Huntington's disease in USD Million (2022-2036)
Table 43: United States Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
Table 44: EU5 Market Size of Huntington's disease in USD Million (2022-2036)
Table 45: EU5 Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
Table 46: Japan Market Size of Huntington's disease in USD Million (2022-2036)
Table 47: Japan Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
List of Figures
Figure 1: Epidemiology and Market Methodology
Figure 2: Part of brain affected by Huntington's Disease
Figure 3: Genetic Mutations in Huntington's Disease
Figure 4: Autosomal Dominant Inheritance
Figure 5: Probability of Inheriting HD
Figure 6: Huntington's Disease Symptoms Triad
Figure 7: Patient Management for Individuals with Huntington's Disease Using the Clinical Practice Guideline
Figure 8: Total Prevalent cases of Huntington's Disease in the 7MM
Figure 9: Total Diagnosed Prevalent cases of Huntington's Disease in the 7MM (2022-2036)
Figure 10: Total Chorea associated cases of Huntington's Disease in the 7MM (2022-2036)
Figure 11: Total Diagnosed Prevalent cases of Huntington's disease by age group in the 7MM (2022-2036)
Figure 12: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in the 7MM (2022-2036)
Figure 13: Total treated cases of Huntington's Disease in the 7MM (2022-2036)
Figure 14: Total Prevalent cases of Huntington's Disease in the US (2022-2036)
Figure 15: Total Diagnosed Prevalent cases of Huntington's Disease in the US (2022-2036)
Figure 16: Total Chorea associated cases of Huntington's Disease in the US (2022-2036)
Figure 17: Total Diagnosed Prevalent cases of Huntington's disease by age group in the US (2022-2036)
Figure 18: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in the US (2022-2036)
Figure 19: Total treated cases of Huntington's Disease in the US (2022-2036)
Figure 20: Total Prevalent cases of Huntington's Disease in the EU5 (2022-2036)
Figure 21: Total Diagnosed Prevalent cases of Huntington's Disease in the EU5 (2022-2036)
Figure 22: Total Chorea associated cases of Huntington's Disease in the EU5 (2022-2036)
Figure 23: Total Diagnosed Prevalent cases of Huntington's disease by age group in the EU5 (2022-2036)
Figure 24: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in the EU5 (2022-2036)
Figure 25: Total treated cases of Huntington's Disease in the EU5 (2022-2036)
Figure 26: Total Prevalent cases of Huntington's Disease in Japan (2022-2036)
Figure 27: Total Diagnosed Prevalent cases of Huntington's Disease in Japan (2022-2036)
Figure 28: Total Chorea associated cases of Huntington's Disease in Japan (2022-2036)
Figure 29: Total Diagnosed Prevalent cases of Huntington's disease by age group in Japan (2022-2036)
Figure 30: Total Diagnosed Prevalent cases of Huntington's disease by Clinical Stage in Japan (2022-2036)
Figure 31: Total treated cases of Huntington's Disease in Japan (2022-2036)
Figure 32: Total Market Size of Huntington's disease in the 7MM, USD Million (2022-2036)
Figure 33: 7MM Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
Figure 34: Total Market Size of Huntington's disease in the US, USD Million (2022-2036)
Figure 35: United States Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
Figure 36: Total Market Size of Huntington's disease in the EU5, USD Million (2022-2036)
Figure 37: EU5 Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
Figure 38: Total Market Size of Huntington's disease in Japan, USD Million (2022-2036)
Figure 39: Japan Market Size of Huntington's disease by Therapies in USD Million (2022-2036)
A selection of companies mentioned in this report includes, but is not limited to:

  • Teva Pharmaceuticals
  • Neurocrine Biosciences
  • Novartis Pharmaceuticals
  • Skyhawk Therapeutics
  • Alnylam Pharmaceuticals
  • Roche
  • UniQure Biopharma

For more information about this report visit

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