Pipeline Insight: Orphan Diseases in CNS - Part I: Huntington's Disease

Publication Date	November 2007
Publisher	Datamonitor
Product Type	Report
Pages	94
ISBN Number	not applicable
Product Code	DAT08406

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Summary

Introduction

The current Huntington's disease market is comprised of off-label prescriptions of psychotropic drugs. In the near term, two pipeline drugs are set to enter the market, demonstrating the viability of this disease as a primary marketing indication. In the longer term, with a strong genetic link and reliable diagnostic test, prophylactic treatment with a disease modifying drug could prove lucrative.

Scope

Scenario based analysis and forecasts of the commercial potential of the Huntington's disease market to 2016.
Analysis of patient potential, marketing factors, commercial attractiveness and clinical attractiveness of key late-stage pipeline drugs.
Strategic recommendations for maximizing the potential of drugs in this niche orphan disease market.
Indication-specific sales forecasts of key pipeline drugs to 2016.

Highlights

NeuroSearch's ACR16 is set to become the market leader by 2010. Targeting a broad spectrum of symptoms, and possessing a novel mode of action, neurologists are likely to view this drug favorably when compared to current treatments.

The launch of Xenazine (tetrabenazine) on the US market and further roll-out across the 5EU looks set to be a good strategic move by Prestwick Pharmaceuticals and Cambridge Laboratories. However, peak market penetration is limited by the drug's use predominantly in severe patients and its poor side-effect profile.

Forecasts based on various potential patient population sizes and drug prices, identify several scenarios in which blockbuster drugs could be established in this market. A theoretical drug which could achieve this is defined.

Reasons to Purchase

Access the lucrative potential of this underdeveloped neurodegenerative disease therapeutic market.
Benchmark key late-stage compounds against current leading therapies and a theoretical "ideal" treatment.
Understand the opportunities in this market based on the comments of interviewed key opinion leaders and industry executives.

Content

About Datamonitor Healthcare
- About The Cns Pharmaceutical Analysis Team
Chapter 1 Executive Summary
- Scope Of The Analysis
- Contributing Experts
- Interviewed Key Opinion Leaders
- Datamonitor Insight Into The Huntington's Disease Market
- Key Metrics
- Datamonitor Pipeline Assessment Summary
- Related Reports
- Upcoming Reports
Chapter 2 Market Overview
- Definition Of Huntington's Disease
- Current Market Overview
- Current Therapies Are Limited To Managing The Symptoms Associated With Hd And Co-Morbidities
- No Treatment Proven To Alter The Course Of The Disease
- Current Treatment Classes
- Current Market Value And Trends
- Antipsychotics And Monoamine-Depleting Agents
- Antidepressants And Anxiolytics
- Anti-Parkinsonism Drugs
- Etiology Of Huntington's Disease
- Risk Of Inheriting Hd
- Prognosis For Hd Patients
- Epidemiology Of Huntington's Disease
- Base Scenario
- High Prevalence Rate Patient Population Scenario
- Very Low Prevalence In Japan
- Increasing Future Trend In Patient Population
- Unmet Need In Huntington's Disease
- Slowing The Progression Of The Disease
- Reduced Movement Disorders
- Reduced Depression, Anxiety And Psychosis
Chapter 3 Future Market Potential
- Future Market Scenarios For Symptomatic Patients
- Base Prevalence Future Market Value Forecast
- High Prevalence Future Market Value
- Price Of Future Hd Drugs
- Current Cost Of Cns Drugs Comparable To A New Disease Modifying Hd Therapy
- Theoretical Disease Modifying Drug
- Pre-Symptomatic "At Risk" Population - The Lucrative Untapped Market
- Scenarios Matrix Summary
Chapter 4 Pipeline Overview And Dynamics
- Pipeline Drugs Market Potential
- Pipeline Overview
- Definition Of Current Comparator Therapy
- Classification Of Pipeline Products
- Recently Discontinued Or Failed Projects
Chapter 5 Late-Stage Drug Analysis & Forecasts
- Xenazine (Tetrabenazine)
- Drug Overview
- Forecasts To 2016
- Datamonitor Drug Assessment Summary
- Marketing Factors
- Market Penetration
- Xenazine Pricing Assumptions
- Patient Potential
- Satisfaction Of Unmet Needs
- Clinical Trial Data
- Tetrabenazine As Antichorea Therapy In Huntington's Disease, Phase Iii Study
- Phase Iii Extension Study
- Older Studies Investigating Tetrabenazine In Hd
- Acr16
- Drug Overview
- Forecasts To 2016
- Datamonitor Drug Assessment Summary
- Marketing Factors
- Market Penetration
- Neurosearch Should Concentrate Marketing Efforts On The Motor Dysfunction Benefits Of Acr16
- Acr16 Pricing Assumptions
- Patient Potential
- The Patient Potential Of Treating Depression
- Positive Effects On Psychosis And Agitation May Also Be Seen In Larger-Scale Trials
- Even If Chorea Is Not Adequately Treated With Acr16, The Patient Potential Would Be High
- Significant Alleviation Of Chorea May Still Be Shown In Larger-Scale Clinical Trials
- Satisfaction Of Unmet Needs
- Clinical Trial Data
- Planned Phase Iii Trials
- Phase Ii Study: Acr16c007
- Pre-Clinical Data - What Is A Dopamine Stabilizer?
- Hd-02 - Ultra-Pure Creatine
- Drug Overview
- Forecasts To 2016
- Marketing Factors
- Patient Potential
- Satisfaction Of Unmet Needs
- Clinical Trial Data
- Planned Phase Iii Trial
- Phase I/Ii Study
- Independent Study On Creatine
- Dimebon
- Drug Overview
- Forecasts To 2016
- Clinical Trial Data
- Ongoing Phase Ii Trial
- Completed Phase I Study
- Further Development And Submission
Bibliography
- Journals
- Websites
Appendix A
- Methodology
- Datamonitor Forecast Methodology For The Hd Market
- Epidemiology Forecasts
- Product Forecasts
- Definition Of A Standard Unit
- Contributing Experts
- Interviewed Neurosearch Company Executives
Appendix B
- About Datamonitor
- About Datamonitor Healthcare
- Datamonitor Healthcare's Therapy Area Capabilities
- About The Disease Analysis Team
- Disclaimer
List Of Tables
- Table 1: Hd Base Scenario Prevalence And Patient Population In The 6mm, 2007
- Table 2: Hd High Prevalence Scenario Patient Population In The 6mm, 2007
- Table 3: Hd Prevalence And Patient Population In Japan, 2007
- Table 4: Price Of Comparable Cns Drugs, 2007
- Table 5: Matrix Of Hd Market Value Dependent On Patient Population And Price In The 6mm, 2007
- Table 6: R&D Clinical-Stage Pipeline In Huntington's Disease, 2007
- Table 7: Xenazine Key Assumptions And Events, 2007
- Table 8: Key Clinical And Commercial Attractiveness Factors For Xenazine
- Table 9: Acr16 Key Assumptions And Events, 2007
- Table 10: Key Clinical/Commercial Attractiveness Factors For Acr16
- Table 11: Datamonitor Drug Assessment Parameters
List Of Figures
- Figure 1: Forecast Of Total 6mm Market Size And Key Pipeline Drugs, 2004-2016,
- Figure 2: Drug Assessment Summary For Hd Treatments, 2007
- Figure 3: Total Sales Revenue Of The Six Major Hd Markets By Region, 2003-07
- Figure 4: Total Sales Revenue Of The Six Major Hd Markets By Drug Class, 2003-07
- Figure 5: Proportion Of Hd-Specific Sales Revenue Attributed To Atypical Versus Typical Antipsychotics In The Us, 2004 And 2006
- Figure 6: Proportion Of Hd-Specific Sales Revenue Attributed To Atypical Versus Typical Antipsychotics In The 5eu, 2004 And 2006
- Figure 7: Genetic Risk Of Developing Hd
- Figure 8: Unmet Needs In Hd Treatment, 2007
- Figure 9: Base Prevalence Future Market Value For Hd ($M), Historical Sales 2004-06, Forecast Sales, 2007-2016, 6mm
- Figure 10: High Hd Prevalence Scenario Future Market Value For Hd ($M), Historical Sales 2004-06, Forecast Sales, 2007-2016, 6mm
- Figure 11: Potential Total Hd Market Value ($M), 2004-2016
- Figure 12: Drug Assessment Summary For Theoretical Drug X For The Treatment Of Hd, 2007
- Figure 13: Summary Of Hd Market And Pipeline Drugs Potential ($M), Historical Sales 2004-06, Forecast Sales, 2007-2016
- Figure 14: Xenazine Hd Specific Sales Revenue Forecast ($M), Historical Sales 2004-06, Forecast Sales 2007-2016
- Figure 15: Drug Assessment Summary For Xenazine In Hd, 2007
- Figure 16: Acr16 Hd-Specific Sales Revenue Forecast ($M), Historical Sales 2004-06, Forecast Sales, 2007-2016
- Figure 17: Drug Assessment Summary For Acr16 In Hd, 2007