Huntington’s Disease Market Poised for Transformation Through Gene Therapies and RNA-Targeted Innovations, with the US Accounting for Over 70% of Global Opportunity | DelveInsight Forecast 2034

Huntington’s Disease Market

The United States dominates the Huntington’s disease market, accounting for over 70% of the total market size across the seven major markets (7MM). Huntington’s disease is a rare, inherited, progressive neurodegenerative disorder with no cure, affecting more than 81,000 prevalent cases in the 7MM. Current treatments are symptom-focused, aiming to manage movement disorders, mood changes, and behavioral symptoms, with FDA-approved options including AUSTEDO, XENAZINE, and the recently approved INGREZZA for chorea. Market growth from 2024 to 2034 is expected to be driven by emerging pipeline therapies such as pridopidine and ANX005, which target unmet needs, along with rising prevalence, increased R&D efforts, advancements in genetic testing, and the introduction of innovative treatment approaches.

DelveInsight’s “Huntington’s Disease Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Huntington’s Disease, historical and forecasted epidemiology as well as the Huntington’s Disease market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

To Know in detail about the Huntington’s Disease market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; https://www.delveinsight.com/sample-request/huntingtons-disease-market

Some of the key facts of the Huntington’s Disease Market Report:

• The Huntington’s Disease market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
• In January 2026, Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the submission of its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority. Pending approval, Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic for the treatment of Huntington’s Disease.
• In November 2025, uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington’s disease (HD).
• In September 2025, UniQure reported positive top-line results from its Phase I/II study of AMT-130, a one-time gene therapy for Huntington’s disease (HD). Top-line data provides a rapid summary of key study results at a predetermined timepoint. The update indicates that AMT-130 significantly slows symptom progression and met the trial’s primary endpoint, marking the first time a therapy has demonstrated the ability to alter the course of HD in a clinical setting.
• In June 2025, Skyhawk Therapeutics, Inc., a clinical-stage biotech company focused on developing small molecule therapies that target RNA, has announced the dosing of the first patient in its Phase 2/3 FALCON-HD trial of SKY-0515. This investigational oral RNA splicing modulator is being evaluated for the treatment of Huntington’s disease (HD). SKY-0515 is designed to lower the levels of HTT and PMS1 proteins, both of which contribute significantly to HD progression. In a Phase 1 trial involving healthy volunteers, the therapy showed a dose-dependent reduction in HTT mRNA, with an average 72% decrease at the highest dose tested, and was generally well tolerated. The Phase 1 study in HD patients, initiated in January 2025, also completed enrollment ahead of schedule in March 2025.
• In December 2024, Novartis entered into an exclusive licensing agreement with PTC Therapeutics to develop and commercialize PTC518, a novel small molecule aimed at targeting the underlying cause of Huntington’s disease. This collaboration represents a strategic initiative by Novartis to advance treatment options in the neurodegenerative disease space, particularly for Huntington’s disease, which currently lacks approved therapies capable of stopping or reversing its progression.
• In September 2024, Skyhawk Therapeutics, Inc., a clinical-stage biotech company focused on developing innovative small molecule therapies targeting RNA, announced that additional positive topline results from Parts A and B of its Phase 1 clinical trial of SKY-0515 will be presented at the European Huntington’s Disease Network and Enroll-HD 2024 meeting, taking place from September 12-14, 2024, in Strasbourg, France.
• In September 2024, Vico Therapeutics, a clinical-stage genetic medicines company, announced positive interim results from its ongoing Phase I/IIa trial of VO659, an investigational treatment for Huntington’s disease. The findings indicate a notable reduction in mutant huntingtin protein levels in the cerebrospinal fluid of treated patients. VO659 is an allele-preferential antisense oligonucleotide (ASO) therapy designed to target the CAG repeat expansion responsible for Huntington’s and other polyglutamine disorders.
• In July 2024, uniQure N.V. (NASDAQ: QURE), a prominent gene therapy company dedicated to developing breakthrough treatments for patients with serious medical conditions, released updated interim data from its ongoing Phase I/II clinical trials of AMT-130 for Huntington’s disease. The data includes follow-up results of up to 24 months from 29 treated patients across the U.S. and Europe.
• In the 7MM population, there will be 81,000 cases of Huntington’s disease overall in 2022. In the 7MM, the US had the highest number of frequently occurring cases of Huntington’s disease, followed by Germany
• In Germany, there were correspondingly 300, 700, 1,200, and 2,000 cases of Huntington’s disease among people aged 20 to 39, 40 to 49, 50 to 59, and 60 or older in 2022.
• In the 7MM, there were 39,000 treated instances of Huntington’s disease overall in 2022; however, this number could rise during the projection period (2023–2022)
• Key Huntington’s Disease Companies: Teva Pharmaceutical (NYSE: TEVA), Annexon Biosciences (NASDAQ: ANNX), Prilenia Therapeutics (Private: N/A), Neurocrine Biosciences (NASDAQ: NBIX), SOM Biotech (Private: N/A), Vaccinex (NASDAQ: VCNX), Sage Therapeutics (NASDAQ: SAGE), uniQure Biopharma (NASDAQ: QURE), Roche (SIX: ROG), Ionis Pharmaceuticals (NASDAQ: IONS), Azevan Pharmaceuticals (Private: N/A), Wave Life Sciences (NASDAQ: WVE), Luye Pharma Group (HKEX: 2186), SOLA Biosciences (Private: N/A), and others
• Key Huntington’s Disease Therapies: AUSTEDO (deutetrabenazine/SD-809), ANX005, Pridopidine(ACR-16; Huntexil), Ingrezza (valbenazine), SOM3355(bevantolol), ANX005, VX15/2503 (pepinemab), SAGE-718, AMT-130/ intrastriatal rAAV5-miHTT, RO7234292, Tominersen, Valbenazine, Pepinemab, SRX246, WVE-003, LPM3770164, SOL-175, and others
• The Huntington’s Disease market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Huntington’s Disease pipeline products will significantly revolutionize the Huntington’s Disease market dynamics.

Huntington’s Disease Overview

Huntington’s disease (HD) is a progressive and inherited neurological disorder that affects the brain’s nerve cells, leading to gradual deterioration of cognitive, motor, and psychiatric functions. HD is caused by a mutation in the HTT gene, which produces a protein called huntingtin. The mutation leads to the production of an abnormal form of the huntingtin protein, which accumulates in neurons and disrupts their function.

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Huntington’s Disease Epidemiology

Huntington’s disease is most prevalent among the geriatric population (aged ≥60 years), with over 21,000 cases reported across the seven major markets (7MM). In 2023, the United States accounted for more than half of the total diagnosed prevalent cases in the 7MM. Within EU4 and the UK, Germany recorded the highest number of Huntington’s disease cases. Additionally, Stage II disease constituted the largest share of Huntington’s disease cases across the 7MM during the same period.

Huntington’s Disease Epidemiology Segmentation:

The Huntington’s Disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Prevalence of Huntington’s Disease
• Prevalent Cases of Huntington’s Disease by severity
• Gender-specific Prevalence of Huntington’s Disease
• Diagnosed Cases of Episodic and Chronic Huntington’s Disease

Download the report to understand which factors are driving Huntington’s Disease epidemiology trends @ Huntington’s Disease Epidemiology Forecast

Huntington’s Disease Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Huntington’s Disease market or expected to get launched during the study period. The analysis covers Huntington’s Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Huntington’s Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Huntington’s Disease Therapies and Key Companies

• AUSTEDO (deutetrabenazine/SD-809): Teva Pharmaceutical
• ANX005: Annexon Biosciences
• Pridopidine(ACR-16; Huntexil): Prilenia Therapeutics
• Ingrezza (valbenazine): Neurocrine Biosciences
• SOM3355(bevantolol): Som Biotech
• ANX005: Annexon Biosciences
• VX15/2503 (pepinemab): Vaccinex
• SAGE-718: Sage Therapeutics
• AMT-130/ intrastriatal rAAV5-miHTT: UniQure Biopharma
• RO7234292: Roche
• Tominersen: Ionis Pharmaceuticals
• Valbenazine: Neurocrine Biosciences
• Pepinemab: Vaccinex
• SRX246: Azevan Pharmaceuticals
• WVE-003: WaVe life Sciences
• LPM3770164: Luye Pharma Group
• SOL-175: SOLA Biosciences

Discover more about therapies set to grab major Huntington’s Disease market share @ Huntington’s Disease Treatment Market

Huntington’s Disease Market Strengths

• As adult-onset Huntington’s disease is more common than juvenile Huntington’s disease, so with growing geriatricpopulation more elderly patients will be diagnosed with HD knowledge

Huntington’s Disease Market Opportunities

• Huntington’s disease is a rare condition thus, companies developing treatment options for the same can possess several advantages like premium pricing, an Orphan Designation, which comes along with 7 years of market exclusivity in the US, clinical trial subsidies, reduced regulatory fees, and several other benefits.

Scope of the Huntington’s Disease Market Report

• Study Period: 2020–2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Huntington’s Disease Companies: Teva Pharmaceutical, Annexon Biosciences, Prilenia Therapeutics, Neurocrine Biosciences, Som Biotech, Annexon Biosciences, Vaccinex, Sage Therapeutics, UniQure Biopharma, Roche, Ionis Pharmaceuticals, Neurocrine Biosciences, Vaccinex, Azevan Pharmaceuticals, WaVe life Sciences, Luye Pharma Group, SOLA Biosciences, and others
• Key Huntington’s Disease Therapies: AUSTEDO (deutetrabenazine/SD-809), ANX005, Pridopidine(ACR-16; Huntexil), Ingrezza (valbenazine), SOM3355(bevantolol), ANX005, VX15/2503 (pepinemab), SAGE-718, AMT-130/ intrastriatal rAAV5-miHTT, RO7234292, Tominersen, Valbenazine, Pepinemab, SRX246, WVE-003, LPM3770164, SOL-175, and others
• Huntington’s Disease Therapeutic Assessment: Huntington’s Disease current marketed and Huntington’s Disease emerging therapies
• Huntington’s Disease Market Dynamics: Huntington’s Disease market drivers and Huntington’s Disease market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Huntington’s Disease Unmet Needs, KOL’s views, Analyst’s views, Huntington’s Disease Market Access and Reimbursement

To know more about Huntington’s Disease companies working in the treatment market, visit @ Huntington’s Disease Clinical Trials and Therapeutic Assessment

Table of Contents

1. Huntington’s Disease Market Report Introduction

2. Executive Summary for Huntington’s Disease

3. SWOT analysis of Huntington’s Disease

4. Huntington’s Disease Patient Share (%) Overview at a Glance

5. Huntington’s Disease Market Overview at a Glance

6. Huntington’s Disease Disease Background and Overview

7. Huntington’s Disease Epidemiology and Patient Population

8. Country-Specific Patient Population of Huntington’s Disease

9. Huntington’s Disease Current Treatment and Medical Practices

10. Huntington’s Disease Unmet Needs

11. Huntington’s Disease Emerging Therapies

12. Huntington’s Disease Market Outlook

13. Country-Wise Huntington’s Disease Market Analysis (2020–2034)

14. Huntington’s Disease Market Access and Reimbursement of Therapies

15. Huntington’s Disease Market Drivers

16. Huntington’s Disease Market Barriers

17. Huntington’s Disease Appendix

18. Huntington’s Disease Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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