Huntington's Disease Market
Key Highlights:
- Huntington’s disease (HD) is an incurable, rare genetic, progressive neurodegenerative disorder that causes death of brain cells, termed as neurons, in several areas of the brain, comprising those that help control voluntary (intentional) movement.
- As per DelveInsight’s estimates, the total prevalent cases of Huntington’s disease were more than 81,000 in seven major markets.
- The treatment for Huntington’s disease is complex as there exists no cure for Huntington’s disease, and there are no treatments known to slow or reverse its process. The current treatment available is symptoms specific, with the treatment goal to slow down the course of the disease and help affected people function for as long and as comfortably as possible.
- Currently, the treatment strategies include the usage of many medications to treat specific symptoms such as medicines for depression, mood swings, and involuntary/abnormal movements and behaviors. The US FDA has approved AUSTEDO (deutetrabenazine) and XENAZINE (tetrabenazine) for the treatment of chorea associated with Huntington’s disease.
- Recently, in August 2023, the US FDA approved INGREZZA (valbenazine) capsules for the treatment of adults with chorea associated with Huntington's disease.
- The United States accounted for the largest market size (more than 70% of the 7MM) of Huntington’s disease, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
- During the forecast period (2024–2034), pipeline candidates such as pridopidine, ANX005, and others are expected to drive the growth of Huntington’s disease treatment market size. Some of these treatments are addressing important areas of unmet medical need, i.e., lack of disease-modifying options for the treatment.
- The growth of the Huntington’s disease market is expected to be mainly driven by entry of innovative emerging therapies, rising prevalence, increasing initiatives for R&D, and the advent of genetic testing.
DelveInsight's “Huntington’s Disease – Market Insights, Epidemiology and Market Forecast – 2034” report delivers an in-depth understanding of Huntington’s disease, historical and forecasted epidemiology as well as the Huntington’s disease market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
Huntington’s disease market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM Huntington’s disease market size from 2020 to 2034. The report also covers current Huntington’s disease treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.
Geography Covered
- The United States
- EU4 (Germany, France, Italy, and Spain) and the United Kingdom
- Japan
Huntington’s Disease Understanding and Treatment Algorithm
Huntington’s Disease Overview, Country-Specific Treatment Guidelines and Diagnosis
Huntington’s disease is inherited as an autosomal dominant trait. In dominant disorders, a single copy of the disease gene (received from either the mother or father) will be expressed “dominating” the other normal gene, resulting in the appearance of the disease. Huntington’s disease is caused by variations (mutations) of a gene (huntingtin) that is located on the short arm (p) of chromosome 4 (4p16.3).
The diagnosis of Huntington’s disease is based on a general physical exam, a review of the patient’s family medical history, as well as neurological, psychiatric examinations, and genetic testing.
The Huntington’s disease report provides an overview of Huntington’s disease pathophysiology, diagnostic approaches, and detailed treatment algorithm along with a real-world scenario of a patient’s journey beginning from the first symptom, the time taken for diagnosis to the entire treatment process.
Further details related to country-based variations in diagnosis are provided in the report
Huntington’s Disease Treatment
Currently, the treatment strategies include the usage of many medications to treat specific symptoms such as medicines for depression, mood swings, and involuntary/abnormal movements and behaviors.
Deutetrabenazine (Brand name: AUSTEDO) and tetrabenazine (Brand name: XENAZINE) and valbenazine (Brand name: INGREZZA) are the US FDA-approved therapies, but their approved indication is the treatment of chorea associated with Huntington’s disease.
Dopamine blockers and drugs such as amantadine can be used to help control involuntary movements and some of the abnormal behaviors associated with this disease. Antidepressants and antipsychotic drugs are prescribed to help with emotional and behavioral problems. Antidepressants, mainly selective serotonin reuptake inhibitors like Lexapro, Prozac, and Zoloft, are frequently used to treat Huntington’s patients’ depression.
Further details related to country-based variations in treatment are provided in the report
Huntington’s Disease Epidemiology
The Huntington’s disease epidemiology chapter in the report provides historical as well as forecasted in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. The Huntington’s disease epidemiology is segmented with detailed insights into Total Prevalent Cases of Huntington’s Disease, Total Diagnosed Prevalent Cases of Huntington’s Disease, Total Chorea Associated Cases of Huntington’s Disease, Total Diagnosed Prevalent Cases of Huntington’s Disease by Age Group, Total Diagnosed Prevalent Cases of Huntington's Disease by Clinical Stage, and Total Treated Cases of Huntington’s Disease.
- According to the findings, Huntington’s disease was more common in geriatric population (≥60 years). In the 7MM, more than 21,000 prevalent cases were observed in people aged more than 60 years.
- As per DelveInsight's estimates, total diagnosed prevalent cases of Huntington’s disease in the United States was more than 50% of total cases in the 7MM in 2023.
- Among EU4 and the UK, the highest number of Huntington’s disease cases was observed in Germany in 2023.
- DelveInsight’s consultant estimates that the Stage II represented majority of the cases for Huntington’s disease in the 7MM in 2023.
Huntington’s Disease Recent Developments
- In February 2025, MMJ International Holdings, a leader in cannabis-derived drug development, reached a key milestone in its efforts to bring MMJ-002—a proprietary soft gelatin capsule combining THC, CBD, and other phytocannabinoids—to market for Huntington's disease.
Huntington’s Disease Drug Chapters
The drug chapter segment of the Huntington’s disease report encloses a detailed analysis of Huntington’s disease marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the Huntington’s disease pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.
Marketed Drugs
AUSTEDO (deutetrabenazine/SD-809): Teva Pharmaceutical
Austedo (deutetrabenazine) is a deuterated form of a small molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that is designed to regulate the levels of a specific neurotransmitter, dopamine, in the brain. It is indicated in adults for the treatment of:
- Chorea associated with Huntington’s disease
- Tardive dyskinesia
The drug has been granted Orphan Drug Designation for the treatment of Huntington’s disease by the US FDA.
INGREZZA (valbenazine/NBI-98854): Neurocrine Biosciences
INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, believed to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function. In August 2023, the FDA granted approval for INGREZZA (valbenazine) capsules to treat chorea in adults with Huntington's disease. The FDA approval is supported by data from two clinical studies conducted in collaboration with the Huntington Study Group (HSG), including the KINECT-HD Phase III study and the ongoing KINECT-HD2 open-label extension trial.
Note: Detailed marketed therapies assessment will be provided in the final report.
Emerging Drugs
Pridopidine (ACR-16): Prilenia Therapeutics
Pridopidine (formerly Huntexil) is Prilenia Therapeutics’ lead asset and a first-in-class selective and potent Sigma-1-receptor (S1R) agonist with neuroprotective properties. Activation of the S1R by pridopidine enhances the clearance of toxic proteins, enhances energy production, and reduces cellular stress and inflammation. These mechanisms are crucial for a neuron’s function and survival. It is a small molecule that is administered in a small easy-to-swallow capsule twice a day. Pridopidine is under late-stage development for the treatment of Huntington's disease (HD).
ANX005: Annexon Biosciences
ANX005 is a clinical-stage investigational monoclonal antibody intended to treat patients with antibody-mediated autoimmune and complement-mediated neurodegenerative disorders. This novel therapy is formulated for IV administration and is designed to inhibit C1q and the entire classical complement pathway. Currently, it is prepared for progressing into the late-stage Phase IIb/III clinical development.
Note: Detailed emerging therapies assessment will be provided in the final report.
Drugs | Company | Molecule Type | MOA | ROA | Phase |
Pridopidine (ACR-16, Huntexil) | Prilenia Therapeutics | Small molecule | Sigma-1 receptor agonist | Oral | III |
Huntington’s Disease Market Outlook
Key players, such as Prilenia Therapeutics, Annexon Biosciences, and others are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of Huntington’s disease.
The United States represents more than 70% of the market size for Huntington’s disease, surpassing the combined market size of Germany, Spain, Italy, France (referred to as EU4), the United Kingdom, and Japan.
- During the forecast period (2024–2034), pipeline candidates such as pridopidine, ANX005, and others are expected to drive the growth of Huntington’s disease market size.
- By 2034, INGREZZA (valbenazine) is anticipated to achieve the prominent market share in the 7 major markets.
- Germany is expected to account for the second highest market size in the 7MM during the forecast period 2024–2034.
Huntington’s Disease Recent Developments
- In December 2024, uniQure N.V. (QURE) saw a 109.7% surge after announcing it had reached an agreement with the FDA on key aspects of an accelerated approval pathway for AMT-130, a one-time investigational gene therapy being developed for the treatment of Huntington’s disease.
- In December 2024, the FDA agreed that results from two ongoing Phase 1/2 clinical trials, compared with an external control group of untreated patients, may be sufficient to support an application for accelerated approval of AMT-130 for Huntington's disease, according to the treatment’s developer.
Huntington’s Disease Drugs Uptake
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024–2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.
Further detailed analysis of emerging therapies drug uptake in the report…
Huntington’s Disease Activities
The report provides insights into different therapeutic candidates in Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Huntington’s disease emerging therapies.
KOL Views
To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.
DelveInsight’s analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as Massachusetts General Hospital, University Hospital of Münster, National Center of Neurology and Psychiatry, etc., were contacted. Their opinion helps understand and validate current and emerging treatment patterns of Huntington’s disease. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. In efficacy, the trial’s primary and secondary outcome measures are evaluated.
Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Market Access and Reimbursement
Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers’ concerns about budget impact. The high cost of rare disease drugs usually has a limited effect on the budget due to the small number of eligible patients being prescribed the drug. The US FDA has approved several rare disease therapies in recent years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products independently.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
Scope of the Report
- The report covers a segment of key events, an executive summary, descriptive overview of Huntington’s disease, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
- Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression along with country specific treatment guidelines.
- Additionally, an all-inclusive account of both the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
- A detailed review of the Huntington’s disease market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
- The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM Huntington’s disease market.
Huntington’s Disease Report Insights
- Patient Population
- Therapeutic Approaches
- Huntington’s Disease Pipeline Analysis
- Huntington’s Disease Market Size and Trends
- Existing and Future Market Opportunity
Huntington’s Disease Report Key Strengths
- Eleven-year Forecast
- 7MM Coverage
- Huntington’s Disease Epidemiology Segmentation
- Inclusion of Country Specific Treatment Guidelines
- KOL’s Feedback on Approved and Emerging Therapies
- Key Cross Competition
- Conjoint Analysis
- Drugs Uptake and Key Market Forecast Assumptions
Huntington’s Disease Report Assessment
- Current Treatment Practices
- Unmet Needs
- Pipeline Product Profiles
- Market Attractiveness
- Qualitative Analysis (SWOT and Conjoint Analysis)
FAQs
- What is the growth rate of the 7MM Huntington’s disease treatment market?
- What was the Huntington’s disease total market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors/key catalysts for this growth?
- Is there any unexplored patient setting that can open the window for growth in the future?
- What are the pricing variations among different geographies for approved and off-label therapies?
- How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
- What are the current and emerging options for the treatment of Huntington’s disease?
- How many companies are developing therapies for the treatment of Huntington’s disease?
- What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
- Patient/physician acceptability in terms of preferred treatment options as per real-world scenarios?
- What are the country-specific accessibility issues of expensive, recently approved therapies?
Reasons to buy
- The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Huntington’s disease market.
- Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
- Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
- Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
- Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
- Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
- Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
- To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
- Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.