United States Huntington's Disease Treatment Market Assessment, By Drug Type [Approved Drugs, Off-Label Drugs], By Distribution Channel [Hospital Pharmacies, Online Pharmacies, Retail Pharmacies], By Region, Opportunities and Forecast, 2018-2032F

United States Huntington's disease treatment market is projected to witness a CAGR of 10.56% during the forecast period, 2025-2032, growing from USD 164.59 million in 2024 to USD 367.44 million in 2032. The United States Huntington's disease treatment market is transforming, and research centers, pharmaceutical companies, and regulatory bodies are intensifying their efforts to bring effective treatments to the market.

The U.S. Huntington's disease treatment market is experiencing steady growth due to rising awareness, early diagnosis, and advancements in therapeutic research. As a rare neurologic disorder, Huntington's disease has also increased demand for treatments for these symptoms, as well as disease-modifying agents for overall improved disease progression. The U.S. healthcare structure has afforded patients access to early genetic testing and complete medical care, which has improved patient treatment outcomes. In challenging drug development, most current therapeutic options for Huntington's disease are primarily based on the symptomatic movement disorder approach. Gene therapies, including RNA-based therapies, and neuroprotective agents now offer a completely new opportunity for treating Huntington's disease. Robust partnerships between academia and the pharmaceutical industry continue to create opportunities for innovation through clinical trials. New treatment options also benefit from regulatory processes for rare disease designations and fast-track review processes. While hospital pharmacies still represent the primary distribution point, access for patients through online pharmacies and convenience factors offer alternative options for access, albeit in a limited manner, through specialty pharmacies.

For instance, in May 2025, according to the American Brain Foundation research, about 1 in 10,000 people in the United States are affected by HD disease, and there is a 50% chance of passing HD disease from parents to their children. Such rising cases of HD (Huntington's Disease) boost the demand for the HD treatment market.

Rising Prevalence of Huntington's Disease Boosts Market Demand

The rising prevalence of Huntington's disease significantly plays a role in increasing market demand for its treatment options. The increasing number of people diagnosed due to improved genetic testing and disease recognition creates an expanding need for effective treatment solutions. Medical professionals and patients search for therapies that manage symptoms along with potential methods to reduce the disease because no cure exists for Huntington's disease. The medical community identifies this disease at its early phases, which enables them to begin therapeutic interventions alongside extended healthcare planning. The growing patient population that requires medical attention creates a greater demand for existing symptom management drugs, as well as new therapies that modify disease progression. Family members of Huntington's disease patients undergo preventive testing because the disease is hereditary, which leads to more diagnoses. The increasing number of patients presents challenges for the healthcare system in delivering advanced, accessible, and effective treatments, thereby driving pharmaceutical advancements and market growth in the U.S. Huntington's disease treatment sector.

For instance, in May 2025, during #LetsTalkAboutHD awareness month, NISOA supported the Huntington's Disease Society of America's (HDSA) efforts to raise awareness about the fight against Huntington's disease.

Advances in Gene and RNA-based Therapies Support Market Expansion

The Huntington's disease treatment market is experiencing transformative expansion due to recent developments in gene and RNA-based therapeutic approaches. The new methods address the root causes of the disease by working to minimize or stop the production of mutant huntingtin protein, rather than providing symptom relief. Antisense oligonucleotides (ASOs), RNA interference (RNAi), and gene editing tools, including CRISPR, undergo clinical testing to modify disease progression. Various companies direct their investments toward these platforms due to the beneficial regulatory pathways, including orphan drug designation and fast-track approvals. Strategic partnerships and funding have been established in response to promising early-phase trial results, which accelerate development timelines. The therapies currently approaching market availability will transition the treatment approach from symptom management to disease alteration. Healthcare providers, along with researchers and patients, are showing growing interest in this evolution, which is expanding the U.S. Huntington's disease treatment market.

For instance, in December 2024, the FDA and uniQure reached an agreement to use an accelerated approval pathway for AMT-130, the company's adeno-associated virus (AAV) vector gene therapy, being assessed in two Phase 1/2 clinical trials for Huntington's disease.

Hospital Pharmacies Hold the Largest Market Share

The U.S. Huntington's disease treatment market receives its most considerable portion from hospital pharmacies, as these facilities are responsible for the care of specialized chronic conditions. Most approved Huntington's disease therapies, which focus on movement disorders and chorea, require hospital-based medical supervision before being administered or prescribed. Hospital pharmacies ensure precise medication dispensing, alongside coordination with neurological specialists and monitoring for side effects, to effectively manage progressive neurodegenerative diseases. Hospital pharmacies provide patients with investigational drugs for access to clinical trials. The combination of diagnostic and treatment services within hospital systems enables efficient patient care, which makes hospital pharmacies the most dependable channel for both providers and patients in this therapeutic field.

Additionally, the pharmaceutical company in the United States increases its sales revenue by selling HD treatment medicines. For instance, in July 2024, Teva Pharmaceutical Industries Ltd. increased its sales revenue in the United States of generic products by approximately 16% to USD 1.02 billion in the second quarter, driven by strong demand for its cheaper copycat medicine and branded treatment for Huntington's disease.

Approved Drugs Dominant the United States Huntington's Disease Treatment Market

Approved drugs dominate the U.S. Huntington's disease treatment market because medical professionals use them extensively, while regulatory bodies approve their use, and they provide immediate symptom control for chorea. The FDA has approved deutetrabenazine and valbenazine, which serve as the conventional treatment for minimizing disease-related involuntary movements. Medical providers select these treatments due to their established safety profiles, proven effectiveness, and reliable results, making them the preferred initial treatment option. Their regulatory approval enables insurance coverage and distribution through hospital and specialty pharmacies, which strengthens their market leadership. Several gene and RNA-based therapies progress through clinical trials, but they remain unavailable for commercial distribution. The Huntington's disease market relies on approved symptomatic treatments to meet patients' immediate needs. The approved drugs maintain their primary status as the most widely used treatment options in U.S. Huntington's disease therapy because of their established clinical use, straightforward prescription process, and extensive market adoption.

For instance, in August 2023, Neurocrine Biosciences, Inc. received approval from the U.S. Food and Drug Administration for INGREZZA (valbenazine) capsules for the treatment of adults with chorea associated with Huntington's disease (HD). INGREZZA is the only selective vesicular monoamine transporter 2 inhibitor that has an effective initial dose, allowing a patient's healthcare provider to modify it based on response and tolerability, with no complex titration required.

Impact of U.S. Tariffs on Huntington's Disease Treatment Market

The U.S. imposes tariffs on pharmaceutical ingredients and biotechnological components, as well as medical equipment, which can substantially impact the Huntington's disease treatment market. Various therapeutic developments depend on obtaining their raw materials and particular equipment from international sources. The imposition of tariffs on imported goods leads to higher production expenses, along with supply chain problems, which extend research and production schedules. Innovative treatments based on gene and RNA technology face particular challenges because their precise materials usually come from international sources. The combination of pricing pressures and reduced investment capacity from companies may result in slower availability of new therapies. Patients will experience increased medication costs and restricted access to advanced medical solutions. The financial obstacles, together with supply chain difficulties caused by tariffs, prevent advancements in this specialized treatment market.

Key Players Landscape and Outlook

Market players are actively expanding their research portfolios through strategic partnerships to accelerate drug development processes. Companies in the biotechnology and pharmaceutical industries direct funds toward gene-editing technologies, along with RNA-targeting therapies and neuroprotective agents, to develop better, long-lasting treatment options. Multiple firms choose to establish licensing agreements and research partnerships with academic institutions, as well as biotech startups, to acquire new platforms and expert knowledge. Companies conduct global clinical trials while applying for Orphan Drug and Fast Track regulatory designations to accelerate development periods. Marketing approaches now focus on launching educational initiatives to increase understanding among healthcare professionals and patients. Huntington's disease treatment market forecast predicts that these coordinated efforts will strengthen competitive positioning while maintaining continuous innovation in treatment approaches.

In May 2025, PTC Therapeutic, Inc. announced that the result of the PTC518 PIVOT-HD study met its primary endpoint, demonstrating a dose-dependent lowering of blood Huntingtin protein at 12 weeks and positive clinical indications at both 12- and 24-months in patients with Stage 2 Huntington's disease, with a favorable tolerability and safety profile overall.