中枢神経系におけるオーファンドラッグ‐第1部：ハンチントン病

Abstract

Overview

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.

Report 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.

Table of Contents

• 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