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Market Spotlight: Huntington's Disease

  • ID: 4421381
  • Report
  • January 2020
  • Region: Global
  • 36 Pages
  • Datamonitor Healthcare
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Huntington’s disease (HD) is a rare disorder of the central nervous system (CNS) which causes the degeneration of nerve cells. It is an autosomal dominant disease characterized by involuntary choreatic movements, psychiatric and behavioral disturbances, and dementia. The mean age of symptom onset is 30-50 years. HD is caused by the presence of 40 cytosine-adenine-guanine (CAG) repeats within the Huntingtin gene. The length of the CAG repeats is inversely associated with the age of disease onset - the longer the repeat, the earlier the onset.

In the case of juvenile HD (JHD), the length of the repeat is 55 and the disease’s symptoms are seen in patients aged 20 years. Learning disabilities and behavioral disturbances are the first symptoms in patients with JHD.

Key Takeaways
  • The author estimates that in 2017, there were 159,410 prevalent cases of Huntington’s disease (HD) in adults aged 30 years and older worldwide, and forecasts that number to increase to 184,950 prevalent cases by 2026.
  • Northern America is estimated to have the highest disease prevalence (0.0073%), while Asia has the lowest prevalence (0.0004%).
  • Teva’s Austedo and Bausch Health’s Xenazine, which target vesicular monamine transporters, are the only marketed drugs for HD. These drugs are administered via the oral route.
  • The majority of industry-sponsored drugs in active clinical development for HD are in Phase II, with two drugs in Phase III. Therapies in active clinical development for HD focus on targets such as huntingtin, vesicular monamine transporters, p38 MAP kinase, SIRT1, vasopressin receptors, semaphorin 4D/CD100, aryl hydrocarbon receptor, NMDA glutamate receptor, PPAR delta, and PPAR gamma. The majority of the pipeline drugs are administered via the oral route, with the remainder being intrathecal, intravenous, and intracerebral/cerebroventricular formulations.
  • High-impact upcoming events for drugs in the HD space comprise topline Phase Ib/IIa trial results for WVE-120101, topline Phase II trial results for pepinemab and HTT-ASO, and topline Phase III trial results for Ingrezza and HTT-ASO.
  • The overall likelihood of approval of a Phase I neurodegenerative asset is 7.5%, and the average probability a drug advances from Phase III is 47.8%. Drugs, on average, take 11.1 years from Phase I to approval, compared to 9.8 years in the overall neurology space.
  • There have been eight licensing and asset acquisition deals involving HD drugs during 2015-20. The $1,050m exclusive strategic collaboration and option agreement signed in 2018 between AbbVie and Voyager Therapeutics, for the development and commercialization of vectorized antibodies directed against tau for the treatment of Alzheimer's disease and other neurodegenerative diseases, was the largest deal.
  • The distribution of clinical trials across Phase I-IV indicates that the majority of trials for HD have been in the early and midphases of development, with 77% of trials in Phase I-II, and only 23% in Phase III-IV.
  • The US has a substantial lead in the number of HD clinical trials globally, while the UK leads the major EU markets. Clinical trial activity in the HD space is dominated by completed trials. Pfizer and Teva have the highest number of completed clinical trials for HD, with 14 trials each.
  • Pfizer and Teva lead industry sponsors with the highest overall number of clinical trials for HD.
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Key Takeaways

Disease Background
  • Subtypes
  • Treatment of chorea
  • Treatment of parkinsonism
  • Treatment of behavioral and psychiatric dysfunction

Marketed Drugs

Pipeline Drugs

Recent Events and Analyst Opinion
  • Nerventra for HD (July 31, 2018)
Key Upcoming Events

Key Regulatory Events
  • Orphan Designation Awarded In March
Probability of Success

Licensing and Asset Acquisition Deals
  • Alkermes Invests In Novel CNS Targets With Rodin Acquisition
Parent Patents

Revenue Opportunity

Clinical Trial Landscape
  • Sponsors by status
  • Sponsors by phase
  • Prescription information

List of Figures
Figure 1: Trends in prevalent cases of HD, 2017–26
Figure 2: Overview of pipeline drugs for HD in the US
Figure 3: Pipeline drugs for HD, by company
Figure 4: Pipeline drugs for HD, by drug type
Figure 5: Pipeline drugs for HD, by classification
Figure 6: Nerventra for HD (July 31, 2018): Phase II - LEGATO-HD
Figure 7: Key upcoming events in HD
Figure 8: Probability of success in the HD pipeline
Figure 9: Licensing and asset acquisition deals in HD, 2015–20
Figure 10: Parent patents in HD
Figure 11: Clinical trials in HD
Figure 12: Top 10 drugs for clinical trials in HD
Figure 13: Top 10 companies for clinical trials in HD
Figure 14: Trial locations in HD
Figure 15: HD trials status
Figure 16: HD trials sponsors, by phase

List of Tables
Table 1: Prevalent cases of HD, 2017–26
Table 2: Prevalence proportions of HD, 2017–26
Table 3: Marketed drugs for HD
Table 4: Pipeline drugs for HD in the US
Table 5: Nerventra for HD (July 31, 2018)
Table 6: Historical global sales, by drug ($m), 2014–18
Table 7: Forecasted global sales, by drug ($m), 2020–24
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