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市場調查報告書
針對亨丁頓舞蹈症的藥劑開發：活潑產品線的希望與機會
Special Report: Huntington's Disease Drug Development--Hope and Opportunity in an Active Pipeline
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針對亨丁頓舞蹈症的藥劑開發：活潑產品線的希望與機會 是由出版商Decision Resources, Inc.在2010年06月所出版的。
這份英文市場調查報告書包含32 Pages 價格從美金9200起跳。
本報告，彙整了美國以及歐洲的亨丁頓舞蹈症病患的動向、亨丁頓舞蹈症的病因、症狀、現在以及新興的治療方式、2009年美國的亨丁頓舞蹈症治療藥物市場規模等，由下列摘要形式闡述。
報告摘要
簡介
亨丁頓舞蹈症的概要
- 發病、症狀、診斷
- 亨丁頓舞蹈症的基因學
- 病態生理學
流行病學
現在的治療方式
- 針對運動障礙管理的治療方式
- 針對行為表現的治療方式
新興的治療方式
今後展望
附錄A：孤兒藥的研發、市場行銷的獎勵方案
附錄B：參考資訊
附錄B：專家訪談
Abstract
Introduction:
Huntington' s disease (HD), a devastating neurodegenerative disease, is the
subject of growing drug development activity as companies seek to address its
significant unmet needs and capitalize on orphan-drug incentives. Increasing
knowledge about the molecular mechanisms of HD and the characterization of a
decades-long preclinical period have shaped a potentially lucrative drug
market. Currently, sales of HD therapies in the United States are constrained
by a small patient population, a paucity of effective therapies, and
widespread use of generics, but thought-leading researchers and physicians
contend that commercial opportunity awaits drug developers willing to forge a
path toward disease-modifying therapies and novel agents for its most
intractable symptoms.
Questions Answered in This Report:
- HD is a rare genetic disease affecting only a small percentage of the
population. How does the prevalence of HD vary geographically? How do
legislative incentives support commercial viability in this orphandisease
market?
- Only one therapy is approved for HD, and so treatment is largely based on
off-label regimens and has no demonstrated effect on HD' s relentless
progression. How has the approval of Lundbeck' s Xenazine (tetrabenazine)
affected the U.S. HD market? What opportunity remains for developers of novel
symptomatic agents?
- HD is the subject of an active R&D pipeline characterized by novel
approaches to modifying the disease' s progression or at least better
controlling its symptoms. Are the potential benefits of disease-modifying
approaches in HD patients proven or theoretical? What are thought leaders'
opinions about the directions of drug research in HD?
- Deficits in motor and cognitive function, increased behavioral problems,
and neuropathological changes are now well documented in gene-positive HD
patients before the onset of clinically manifest disease. Which
porganizations or companies have jumped into the untested waters of pre-HD
clinical trials? What are the commercial opportunities that incentivize a
paradigm shift in drug development?
Scope:
- Primary research: Interviews with five HD experts in two countries.
- Patient population: General HD population in the United States and
Europe; prevalence estimates for select regions worldwide.
- Potential HD etiology: Aggregation of mutant huntingtin protein,
excitotoxicity, oxidative stress, mitochondrial dysfunction, and other
mechanisms causing neuronal death.
- HD genetic basis, onset, and symptoms: Expression of mutant
huntingtin protein; presentation of motor, cognitive, and behavioral symptoms;
evolving characterization of prodromal/pre-manifest HD.
- Current and emerging HD therapies: Symptomatic treatments
(including the only approved HD therapy, Lundbeck' s Xenazine); glutamate
regulators, dopamine stabilizers, gene therapy, HDAC inhibitors, RNAi,
antisense, and other emerging approaches.
- HD market size: 2009 U.S. HD pharmaceutical sales.
Table of Contents
- Executive Summary
- Strategic Considerations
- Stakeholder Implications
- Introduction
- Overview of Huntington' s Disease
- Disease Onset, Symptoms, and Diagnosis
- Genetics of Huntington' s Disease
- Pathophysiology
- Epidemiology
- Current Therapies
- Therapies for Control of Disordered Movement
- Therapies for Behavioral Manifestations
- Emerging Therapies
- Disease-Modifying Therapies
- Symptomatic Therapies
- Outlook
- Appendix A. Incentive Programs for the Research, Development, and
Marketing of Orphan Drugs
- FDA
- Expanded Market Exclusivity
- Tax Credits
- Support During the Drug Approval Process
- Grants
- European Medicines Agency
- Private Organization Incentives
- Appendix B. Bibliography
- Appendix C. Experts Interviewed - Huntington' s Disease
Tables
- 1. Thought Leaders' Opinions on Huntington' s Disease Issues
- 2. Effect of CAG Repeats on Huntington' s Disease Development
- 3. Select Emerging Therapies for the Treatment of Huntington' s
Disease
- 4. Sales of Agents to Treat Huntington' s Disease in the United
States, 2009
Figures
- 1.Inheritance Patterns in Huntington' s Disease
- 2. Brain Structures Implicated in Huntington' s Disease
- 3. Documented Prevalence of Huntington' s Disease in Select
Geographic Regions
- 4. Timeline of Expected Events in the U.S. Huntington' s Disease
Therapy Market
- A1. Drugs Receiving FDA Orphan-Drug Designation and Approval,
1983-2009
- A2. Growth in the Number of Orphan Drugs Available, 1983-2009
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