huntingtons disease market insight

DelveInsight’s ‘Huntington’s Disease (HD) - Market Insights, Epidemiology, and Market Forecast—2030’ report delivers 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, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The Huntington’s Disease market report provides current treatment practices, emerging drugs, Huntington’s Disease market share of the individual therapies, current and forecasted Huntington’s Disease market size from 2018 to 2030 segmented by seven major markets. The Report also covers current Huntington’s Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2018–2030

Huntington’s Disease Understanding and Treatment Algorithm

Overview

Huntington’s Disease (HD) is a rare, incurable, genetic, progressive neurodegenerative disorder that causes brain cells, termed as neurons, to die in several areas of the brain, together with those that help control voluntary (intentional) movement. It is passed on (inherited) from a person’s parents and known to get worse over time, disturbing the patient’s physical movements, emotions, and cognitive abilities. Huntington’s Disease is named after George Huntington, the physician who described it as hereditary chorea in 1872.

Huntington’s Disease is caused by an error in the DNA commands that build our bodies and keep them running. DNA is made up of thousands of genes, and people with Huntington’s Disease have a small fault in one gene, chromosome 4 – one of the 23 human chromosomes that carry a person’s entire genetic code. This defect is “dominant,” meaning that any person who inherits it from a parent with Huntington’s will develop the disease in the long run. The faulty gene codes the blueprint for a protein called huntingtin. This protein’s normal function is not yet known; however, it is called “huntingtin” because scientists acknowledged its defective form as the cause of Huntington’s Disease. Over time, this defective huntingtin protein leads to brain changes that cause abnormal involuntary movements, a severe decline in thinking and reasoning skills, and irritability, depression, and other mood changes. Huntington’s Disease symptoms can develop at any time, but they often first appear when people are in their 30s or 40s.

Huntington’s Disease is generally characterized by the gradual development of involuntary muscle movements affecting the hands, feet, face, and trunk and progressive weakening of cognitive processes and memory (dementia). Neurologic movement abnormalities may include uncontrolled, irregular, rapid, jerky movements (chorea) and athetosis, a condition characterized by relatively slow, writhing involuntary movements. Dementia is typically associated with progressive disorientation and confusion, personality disintegration, impairment of memory control, restlessness, agitation, and other symptoms and findings. In addition, life-threatening complications may result from pneumonia or other infections, injuries related to falls, or other associated developments.

Huntington’s Disease Diagnosis

A diagnosis of Huntington’s Disease is typically suspected in people with characteristic signs and symptoms of the condition and a family history consistent with autosomal dominant inheritance. Not surprisingly, a family history of the disorder is often the biggest clue that a person may have Huntington’s Disease. Special blood tests can help the health care provider determine an individual’s likelihood of developing Huntington’s Disease. Specialized x-ray studies such as computerized tomography (CT) scanning, magnetic resonance imaging (MRI), or electroencephalography (EEG) may help confirm the diagnosis of Huntington’s Disease.

Huntington’s Disease Treatment

Unfortunately, there is no cure for Huntington’s Disease, and there are no treatments known to slow or reverse its process. No treatments can alter the course of Huntington’s Disease. However, medications can lessen some symptoms of movement and psychiatric disorders. And multiple interventions can help a person adapt to changes in their abilities for a certain amount of time. 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. Depression and suicide are more common among affected people, so caregivers should monitor for associated symptoms and seek help if necessary. It becomes crucial for caregivers to watch symptoms closely and seek medical help immediately when issues or questions come up. As symptoms of the disease worsen, affected people need more assistance, supervision, and care.

Deutetrabenazine (Brand name: Austedo) and tetrabenazine (Brand name: Xenazine) are the only United States Food and Drug Administration (FDA)-approved therapies, but their approved indication is the treatment of chorea associated with Huntington’s Disease. Possible side effects include drowsiness, restlessness, and the risk of worsening or triggering depression or other psychiatric conditions.

Huntington’s Disease Epidemiology

In 2020, the overall prevalent cases of Huntington’s Disease accessed to be around 80,170 in the 7MM, which are expected to grow during the forecast period, i.e., 2021–2030.

The disease epidemiology covered in the report provides historical as well as forecasted Huntington’s Disease epidemiology [segmented as Total prevalent cases of Huntington’s Disease, Total diagnosed cases of Huntington’s Disease, Age-specific cases of Huntington’s Disease, Stage-specific cases of Huntington’s Disease, and Total treated cases of Huntington’s Disease] in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2018 to 2030.

Country Wise- Huntington’s Disease Epidemiology

Key Findings

• In the United States, the total number of prevalent cases of Huntington’s Disease were 42,414 in 2020, which are expected to increase during the forecast period, i.e., 2021–2030.
• In 2020, the total prevalent cases of Huntington’s Disease were 35,930 in EU-5, which are projected to increase during the forecast period, i.e., 2021–2030.
• Findings from various secondary studies suggest that Huntington’s Disease is more prevalent in elderly population.
• In addition, secondary data suggest that less people identified in stage IV and stage V with Huntington's disease. In 2020, 3,195 Stage IV cases of Huntington’s Disease and 777 Stage V cases of Huntington’s Disease were estimated for the 7MM.

Huntington’s Disease Drug Chapters

Drug chapter segment of the Huntington’s Disease report encloses the detailed analysis of Huntington’s Disease developmental stage pipeline drugs. It also helps to understand the Huntington’s Disease clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Huntington’s Disease Marketed Therapies

Austedo (Deutetrabenazine): Teva Pharmaceuticals

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. The FDA granted Deutetrabenazine New Chemical Entity Exclusivity until April 2022 and Orphan Drug exclusivity for the treatment of chorea associated with Huntington disease until April 2024.

Note: Full and detailed list of emerging therapies will be provided in the final report.

Huntington’s Disease Emerging Drugs

Ingrezza (Valbenazine/NBI-98854): Neurocrine Biosciences

Neurocrine Biosciences is currently developing Valbenazine, also known as Ingrezza, an investigational treatment for chorea associated with Huntington’s Disease. It is a selective VMAT2 inhibitor, is believed to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function. At present, this drug is being evaluated in two Phase III (NCT04400331 and NCT04102579) trials. KINECT-HD (NCT04102579) is a multi-center, randomized, double-blind, placebo-controlled study conducted in collaboration with the University of Rochester’s Clinical Trials Coordination Center, and sponsored by Neurocrine Biosciences, a neuroscience-focused biopharmaceutical company that discovered and developed valbenazine.

Pridopidine: Prilenia

Pridopidine, is currently being studied in the ongoing Phase III global clinical trial PROOF-HD (Pridopidine Outcome On Function in Huntington Disease). Recent studies have shown that pridopidine enters the brain and spinal cord, where it activates a protein called the Sigma-1 receptor (S1R). The S1R is highly expressed in the brain where it regulates several cellular mechanisms common to neurodegenerative diseases such as Huntington’s Disease.

ANX005: Annexon

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. ANX005 is currently in Phase II (NCT04514367) for treating patients with Huntington’s Disease.

Pepinemab: Vaccinex

Vaccinex is developing Pepinemab for Huntington’s Disease. Vaccinex has recently completed the SIGNAL trial (NCT02481674), a late-stage clinical study of pepinemab for Huntington’s Disease. Pepinemab works by inhibiting SEMA4D, which controls the activity of several different cell types important to normal brain function.

SRX246: Azevan Pharma

SRX246 is a small molecule, centrally-active, highly-selective vasopressin V1A receptor antagonist which is under investigation by Azevan Pharmaceuticals. The Company completed two Phase II clinical trials in adults with its lead compound, SRX246, for the treatment of Intermittent Explosive Disorder and for the treatment of irritability of Huntington’s Disease patients (the STAIR trial). The National Institute of Neurological Diseases and Stroke awarded the Company a major grant to conduct a Phase II trial with SRX246 in Huntington's disease patients exhibiting irritability, a major neuropsychiatric symptom. The trial was conducted and completed in collaboration with the NINDS NeuroNext network across 22 sites nationwide.

AMT-130: UniQure Biopharma

UniQure is developing a gene therapy for Huntington’s Disease (AMT-130), a rare, fatal, neurodegenerative genetic disorder that affects motor function and leads to behavioral symptoms and cognitive decline in young adults, resulting in total physical and mental deterioration. AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging the proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). The drug has received orphan drug designation and fast track designation from the US FDA for the treatment of Huntington’s Disease and is currently in Phase I/II of development.

Note: Full and detailed list of emerging therapies will be provided in the final report.

Huntington’s Disease Market Outlook

Key Findings

The Huntington’s Disease market size in the 7MM is expected to change during the study period 2018–2030, at a CAGR of 22.5%. According to the estimates, the highest market size of Huntington’s Disease is accessed in the United States followed Germany, and the UK, in 2020.

The United States Market Outlook

In United States, the total market size of Huntington’s Disease is expected to increase at a CAGR of 20.3% during the study period (2018–2030).

EU-5 Countries: Market Outlook

In EU-5, the total market size of Huntington’s Disease is expected to increase at a CAGR of 36.5% during the study period (2018–2030).

Japan Market Outlook

The total market size of Huntington’s Disease in Japan is estimated to rise at a CAGR of 31.4%, during the study period (2018–2030).

Huntington’s Disease Pipeline Development Activities

The drugs which are in pipeline include:

1. Pridopidine (ACR-16): Prilenia Therapeutics
2. Ingrezza (Valbenazine/NBI-98854): Neurocrine Biosciences
3. ANX005: Annexon Biosciences
4. SOM3355/Bevantolol: SOM Biotech
5. SRX246: Azevan Pharmaceuticals
6. Pepinemab (VX15/2503): Vaccinex
7. AMT-130: UniQure Biopharma
8. WVE-003: Wave life sciences/Takeda
9. NP03/Lithium citrate/NanoLithium/NanosiRNA: Medesis Pharma

Note: Detailed emerging therapies assessment will be provided in the final report

Huntington’s Disease Drugs Uptake

Analysts Insight

Currently, there are two medications that are FDA-approved for Huntington’s Disease. Both medicines have been approved to treat Huntington's disease chorea (uncontrollable movements). During the forecast period (2021–2030), pipeline candidates are expected to drive the rise in Huntington’s Disease market size. Some of these treatments are addressing important areas of unmet medical need, i.e., lack of disease-modifying options for the treatment. Wave Life Sciences (WVE-120102 and WVE-120101) and Roche/Ionis (tominersen) both reported disappointing results in Huntington's disease clinical trials. Roche's product has been discontinued, leaving a void in late-stage Huntington's disease trials. After the failure of Wave Life Sciences and Roche/Ionis’s products, Huntington’s Disease community’s eyes are on UniQure’s AMT-130 gene therapy. Apart from this, like tetrabenazine and Austedo (deutetrabenazine), Neurocrine Biosciences' Ingrezza (valbenazine) is being investigated to treat chorea symptoms. In the coming years, Ingrezza is likely to compete directly with Teva's Austedo.

Access and Reimbursement Scenario in Huntington’s Disease Therapies

The Healthwell foundation assists with the prescription drugs and biologics used in the treatment of Huntington’s Disease. Under this fund, HealthWell can assist with premium costs. Medicare supplemental policies can help with cost shares related to many aspects of patient’s health care. The maximum award level is USD 10,000. The treatments covered under the plan are amantadine, austedo, cesamet, co-enzyme Q10, deutetrabenazine, haldol, haldol decanoate, haloperidol, nabilone, Q-sorb Co Q-10, quinzyme, rilutek, riluzole, symmetrel, tetrabenazine, ubiquinone, and xenazine.

Note: This is a Medicare Access Fund. In order to qualify for assistance through this fund, the patient must have Medicare. The Foundation will refer patients without Medicare to other programs, such as manufacturer patient assistance programs. Additionally, HealthWell assists individuals with incomes up to 400-500% of the Federal Poverty Level. The Foundation also considers the number in a household and cost of living in a particular city or state

KOL-Views

To keep up with current market trends, we take KOLs and SME’s opinion working in the Huntington’s Disease domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Huntington’s Disease market trend. This will support the clients in potential upcoming novel treatment by identifying the over Huntington’s Disease scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Huntington’s Disease Market by using various Competitive Intelligence tools that includes – SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Huntington’s Disease, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.
• Comprehensive insight has been provided into the Huntington’s Disease epidemiology and treatment in the 7MM.
• Additionally, an all-inclusive account of both the current and emerging therapies for Huntington’s Disease is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of Huntington’s Disease market; historical and forecasted is included in the report, covering drug outreach in the 7MM.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Huntington’s Disease market.

Report Highlights

• In the coming years, Huntington’s Disease market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Huntington’s Disease R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Huntington’s Disease.
• Our in-depth analysis of the pipeline assets across different stages of development different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the over Huntington’s Disease scenario of the research and development activities.

Huntington’s Disease Report Insights

• Patient Population
• Therapeutic Approaches
• Huntington’s Disease Pipeline Analysis
• Huntington’s Disease Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Huntington’s Disease Report Key Strengths

• 10 Years Forecast
• 7MM Coverage
• Huntington’s Disease Epidemiology Segmentation
• Highly Analyzed Market
• Drugs Uptake

Huntington’s Disease Report Assessment

• SWOT Analysis
• Current Treatment Practices
• Unmet Needs
• Market Attractiveness
• Market Drivers and Barriers

Key Questions

Market Insights:

• What was the Huntington’s Disease Market share (%) distribution in 2018 and how it would look like in 2030?
• What would be the Huntington’s Disease total market size as well as market size by therapies across the 7MM during the study period (2018–2030)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest Huntington’s Disease market size during the study period (2018–2030)?
• At what CAGR, the Huntington’s Disease market is expected to grow in the 7MM during the study period (2018–2030)?
• What would be the Huntington’s Disease market outlook across the 7MM during the study period (2018–2030)?
• What would be the Huntington’s Disease market growth till 2030 and what will be the resultant market size in the year 2030?
• How would the market drivers, barriers and future opportunities affect the market dynamics and a subsequent analysis of the associated trends?
• Huntington’s Disease patient types/pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
• How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration and frequencies on the basis of their clinical trial results?
• Among the emerging therapies, what are the potential therapies which are expected to disrupt the Huntington’s Disease market?

Epidemiology Insights:

• What is the disease risk, burden and unmet needs of the Huntington’s Disease?
• What is the historical Huntington’s Disease patient pool in the seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
• What would be the forecasted patient pool of Huntington’s Disease in the 7 major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
• What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Huntington’s Disease?
• Out of Huntington’s Disease the 7MM countries, which country would have the highest prevalent population of Huntington’s Disease during the study period (2018–2030)?
• At what CAGR the population is expected to grow in the 7MM during the study period (2018–2030)?
• What are the various recent and upcoming events which are expected to improve the diagnosis of Huntington’s Disease?

Current Treatment Scenario and Emerging Therapies:

• What are the current options for the treatment of Huntington’s Disease?
• What are the current treatment guidelines for the treatment of Huntington’s Disease in the US, Europe and Japan?
• How many companies are developing therapies for the treatment of Huntington’s Disease?
• How many therapies are developed by each company for the treatment of Huntington’s Disease?
• How many emerging therapies are in mid stage, and late stage of development for the treatment of Huntington’s Disease?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Huntington’s Disease therapies?
• What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Huntington’s Disease and their status?
• What are the key designations that have been granted for the emerging therapies for Huntington’s Disease?
• What is the global historical and forecasted market of Huntington’s Disease?

Reasons to buy

• The report will help in developing business strategies by understanding trends shaping and driving the Huntington’s Disease market.
• To understand the future market competition in the Huntington’s Disease market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Huntington’s Disease in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
• Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for Huntington’s Disease market.
• To understand the future market competition in the Huntington’s Disease market.