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市場調查報告書

CNS(中樞神經系統)疾病用孤兒藥:R&D及市場

Orphan Drugs for CNS Diseases: R&D and Market 2014-2024

出版商 Visiongain Ltd 商品編碼 293356
出版日期 內容資訊 英文 130 Pages
商品交期: 最快1-2個工作天內
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CNS(中樞神經系統)疾病用孤兒藥:R&D及市場 Orphan Drugs for CNS Diseases: R&D and Market 2014-2024
出版日期: 2014年01月10日 內容資訊: 英文 130 Pages
簡介

全球稀少中樞神經系統疾病用治療藥市場,預計2018年超過25億美元。

本報告提供全球中樞神經系統(CNS)疾病用孤兒藥市場相關調查、銷售額實際成果、技術、R&D趨勢、機會及收益預測等、全球、次市場、產品及主要國家市場分析與預測、產業整體趨勢,及今後的市場展望等彙整資料,為您概述為以下內容。

第1章 摘要整理

第2章 CNS疾病的孤兒藥(稀少藥):簡介

  • 罕見疾病定義
  • 主要的稀少CNS適應症是什麼?
  • 孤兒藥指定:對企業的激勵
  • 氣勢漸增的孤兒藥需求

第3章 全球CNS孤兒藥市場市場

  • 孤兒藥市場規模
  • CNS孤兒藥市場:收益預測
  • 亨丁頓舞蹈症的孤兒藥
  • 肌肉萎縮性側索硬化症(俗稱漸凍人)(ALS)的孤兒藥
  • 重症肌無力的孤兒藥

第4章 稀少CNS適應症的主要國家市場

  • CNS孤兒藥市場的各地區分析
  • 主要國家市場:收益預測
  • 美國:CNS孤兒藥的首位市場
  • 主要歐盟市場的孤兒藥
  • 日本的CNS孤兒藥市場:收益預測
  • 新興國家市場上CNS孤兒藥市場潛力

第5章 主要CNS孤兒藥的展望

  • 亨丁頓舞蹈症治療
  • 肌肉萎縮性側索硬化症(ALS)治療
  • 重症肌無力治療

第6章 CNS孤兒藥:研發產品線

  • 亨丁頓舞蹈症開發平台的治療藥
  • 肌肉萎縮性側索硬化症(ALS)開發平台的治療藥
  • 重症肌無力開發平台的治療藥

第7章 CNS孤兒藥:產業趨勢

  • 優勢和弱點
  • 機會及威脅
  • STEP分析
  • CNS孤兒藥開發趨勢
  • CNS的孤兒藥的商業化

第8章 調查採訪

第9章 結論

  • CNS孤兒藥市場的狀況
  • CNS罕見疾病的再度關心促進市場
  • 美國市場今後10年繼續支配市場
  • 治療費用高將抑制市場潛力

圖表一覽

企業索引

目錄

Uncommon nerve disorders - new study explains technological and commercial prospects

What's the future of medicines for rare central nervous system (CNS) disorders? Visiongain's new report gives you predictions for those therapies. Stay ahead. There you explore sales results, technologies, R&D trends, opportunities, and revenue forecasts.

So see what's possible for treating unusual neurological conditions. Avoid missing out. You gain data and financial analysis for that industry and market, appraising its outlooks. Find technological progress and commercial prospects, seeing potential gains.

Read on to explore those treatments and find what their future could be worth.

Forecasts and other analyses for therapy of rare (orphan) neurodegenerative disorders

Our new study reveals sales predictions to 2024 at overall world, submarket, product, and national level. For those therapies, you investigate where the money lies. Find potentials.

Besides forecasting revenues to 2024, that work shows historical results, growth rates, and market shares. There you get feel for technologies, regulatory outlooks, and developments (R&D). You also gain 41 tables, 40 charts, and two interviews with the industry.

Knowledge to help your work and save time, helping your influence

Our report strives to help your business research, analyses, and decisions on treatments for debilitating CNS diseases. There also see how you can save time. And find how you could benefit your authority through better understanding the present and future.

So the following sections explain what you get in that new investigation.

Forecasting of that world market and its main segments - you explore possibilities

What're the secrets of that industry's progress? What's its potential? Discover in our work overall world revenue to 2024 for orphan-drug CNS therapy.

Also in that report you see individual revenue predictions to 2024 for four therapeutic submarkets at world level:

  • Huntington's disease (HD)
  • Amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease, motor neurone disease)
  • Myasthenia gravis (MG)
  • Other treatment applications.

How will those categories expand? Which classes of therapy can generate most money? So you assess outlooks for sales growth, seeing where you can gain.

There you explore competition and rising sales for treating the rarer abnormalities causing neurodegeneration, autoimmune neuromuscular disorder, and other nerve disease.

You also assess top products' futures.

Predictions of leading agents' revenues

Find sales outlooks for brands, too, seeing how they compete and succeed. Our study shows you individual revenue forecasts to 2024 for four marketed drugs:

  • Xenazine (tetrabenazine)
  • Rilutek (riluzole)
  • Mestinon (pyridostigmine)
  • Imuran (azathioprine).

There you find products and years with highest predicted sales and growth. Also you examine competitors. You see what's happening, then, understanding challenges, competition, opportunities, and potentials.

Our study also divides its overall prediction into geographical regions.

Healthcare in leading national markets - what outlooks for those CNS drug sales?

In developed and developing countries, opportunities for providers of orphan neurological medicines will increase from 2014 to 2024. See where and how, finding sales potentials.

Our analyses show you individual revenue forecasts to 2024 for 11 national markets:

  • United States (US)
  • Japan
  • EU leaders - Germany, France, the UK, Italy, and Spain (EU5)
  • BRIC nations - Brazil, Russia, India, and China.

There you find countries with highest revenues, demand, and potential sales growth. Our work explains. You assess needs and demands for those specialist medicines.

Explore, too, how that neurological pharma technology benefits developers, producers, marketers, and sellers. Our work shows you, discussing issues.

Events and forces for developing, producing, marketing, and selling CNS orphan medicines

Our report shows you issues and events affecting that pharmaceuticals sector from 2014, including these:

  • Candidates in development - R&D pipelines - for those indications
  • Benefits of orphan drug designation
  • Treatment needs - unmet and under-met - stimulating demand
  • Regulatory incentives for developers, producers, and sellers.

And these influences:

  • Drug launches expected and their implications for that industry and market
  • Potential for orphan CNS products in emerging national markets
  • Innovations - including drug-device combinations, neurotrophic factors to reverse disease, cellular therapy, and stimulating of neuronal growth
  • Companion diagnostics and personalised medicine.

There you explore political, economic, social, and technological questions, investigating outlooks for commerce. Discover what the future holds.

Examine, then, what stimulates and restrains players in that industry, and affects results.

Companies and 2018 market value - find prospects for success

What happens next? From 2014 new and novel treatments and regulations there encourage investments, medical advances, and higher revenues.

Our report, then, predicts the world market for drugs treating those rare CNS conditions will exceed $2.5bn in 2018. There big pharma and specialty companies can gain. Explore how.

There you examine activities of many companies, including these:

  • GlaxoSmithKline (GSK)
  • Lundbeck
  • Valeant Pharmaceuticals
  • Teva Pharmaceutical Industries
  • BrainStorm Cell Therapeutics.

R&D in that industry holds strengths, and from 2014 there will arise many opportunities. From this decade, patients, payers, and pharmaceutical companies will benefit.

And you discover how that market can perform, staying ahead in knowledge.

Ways Orphan Drugs for CNS Diseases: R&D and Market 2014-2024 helps you

In particular, our investigation gives you these advantages:

  • Revenues there to 2024 at world level, for 4 submarkets, and 4 top products - examine outlooks for production, marketing, and sales
  • Forecasts to 2024 for 11 national markets in the Americas, Europe, and Asia - assess developed and developing countries for potential revenues
  • Prospects for established competitors, emerging companies, and new entrants - explore portfolios, results, strategies, and developmental activities.

Information found nowhere else, helping your searches, analyses, and plans

That study gives independent analysis. There you receive competitive intelligence found only in our work, finding where progress and money lie. Explore the commercial possibilities.

With that data you're less likely to fall behind in knowledge or miss opportunity. Being free to choose, you find how you could save time and effort with our study, helping your decisions.

Table of Contents

1. Executive Summary

  • 1.1 Orphan Drugs for CNS Diseases: Market Overview
  • 1.2 Report Contents - Chapter Overviews
  • 1.3 Research and Analysis Methods

2. Introduction to Orphan Drugs for CNS Disorders

  • 2.1 Defining Orphan Diseases
  • 2.2 What are the Major Orphan CNS Indications?
    • 2.2.1 Huntington's Disease
    • 2.2.2 Amyotrophic Lateral Sclerosis (ALS)
    • 2.2.3 Myasthenia Gravis
    • 2.2.4 Other Orphan CNS Indications
  • 2.3 Orphan Drug Designation: Providing Incentives to Companies
    • 2.3.1 What Benefits Come with Orphan Drug Designation?
  • 2.4 Demand for Orphan Drugs Gathers Momentum
    • 2.4.1 Development of Orphan Drugs

3. The World Orphan CNS Drugs Market, 2014-2024

  • 3.1 How Large Was the Orphan Drugs Market in 2013?
    • 3.1.1 Huntington's Disease: The Largest Orphan CNS Disease Sector in 2012
  • 3.2 The Orphan CNS Drug Market: Revenue Forecast, 2013-2024
    • 3.2.1 What Will Drive Growth in the Market to 2024?
    • 3.2.2 What Will Restrain the Market From 2014-2024?
  • 3.3 Orphan Drugs in Huntington's Disease
    • 3.3.1 Huntington's Disease: Submarket Forecast, 2013-2024
      • 3.3.1.1 Huge Unmet Need to Drive HD Treatment Market
      • 3.3.1.2 Enactment of New Law in US May Add Growth Impetus
  • 3.4 Orphan Drugs in Amyotrophic Lateral Sclerosis
    • 3.4.1 Amyotrophic Lateral Sclerosis: Submarket Forecast, 2013-2024
      • 3.4.1.1 New Drug Launches Will Provide Market Recovery Following Patent Expiry of Rilutek
  • 3.5 Orphan Drugs in Myasthenic Gravis
    • 3.5.1 Myasthenia Gravis: Submarket Forecast, 2013-2024
      • 3.5.1.1 Clinical R&D into New Treatments Keep Patients Hopeful

4. Leading National Markets for Rare CNS Indications 2014-2024

  • 4.1 Regional Breakdown of The Orphan CNS Drug Market
  • 4.2 Leading National Markets: Revenue Forecasts, 2013-2024
  • 4.3 The US: The Leading Market for Orphan CNS Drugs in 2012
    • 4.3.1 Orphan Drug Regulations in the US
    • 4.3.2 Benefits for Orphan Drug Developers
    • 4.3.3 The US Orphan CNS Drug Market: Revenue Forecast, 2013-2024
  • 4.4 Orphan Drugs in Leading EU Markets
    • 4.4.1 The EU5 Orphan CNS Drug Market: Revenue Forecasting, 2013-2024
      • 4.4.1.1 The Incentives for Orphan Drug Development in the EU
    • 4.4.2 Orphan CNS Drugs in the EU5: Submarket Revenue Forecasts, 2013-2024
      • 4.4.2.1 The German Orphan CNS Drug Market: Revenue Forecast, 2013-2024
      • 4.4.2.2 The French Orphan CNS Drug Market: Revenue Forecast, 2013-2024
      • 4.4.2.3 The UK Orphan CNS Drug Market: Revenue Forecast, 2013-2024
      • 4.4.2.4 The Spanish Orphan CNS Drug Market: Revenue Forecast, 2013-2024
      • 4.4.2.5 The Italian Orphan CNS Drug Market: Revenue Forecast, 2013-2024
  • 4.5 The Japanese Orphan CNS Drug Market: Revenue Forecast, 2013-2024
    • 4.5.1 Orphan Drug Regulations and Benefits in Japan
  • 4.6 Potential for Orphan CNS Drugs in Emerging National Markets, 2013-2024
    • 4.6.1 The Chinese Orphan CNS Drug Market: Revenue Forecast, 2013-2024
    • 4.6.2 The Indian Orphan CNS Drug Market: Revenue Forecast, 2013-2024
    • 4.6.2.1 Large Patient Population to Draw Drug Developers to India
    • 4.6.3 The Russian Orphan CNS Drug Market: Revenue Forecast, 2013-2024
      • 4.6.3.1 Attempts to Introduce Orphan Designation and Incentives 2010-2013
    • 4.6.4 The Brazilian Orphan CNS Drug Market: Revenue Forecast, 2013-2024

5. Outlooks for Leading Orphan CNS Drugs 2014-2024

  • 5.1 Huntington's Disease Treatments
    • 5.1.1 Xenazine (Tetrabenazine; Lundbeck/Valeant Pharmaceuticals)
      • 5.1.1.1 Omerus Pipeline HD Treatment Receives Orphan Designation
      • 5.1.1.2 Xenazine: Revenue Forecast, 2013-2024
  • 5.2 Amyotrophic Lateral Sclerosis Treatments
    • 5.2.1 Rilutek (Riluzole; Covis Pharmaceuticals)
      • 5.2.1.1 Patent Expiry to Cause Influx of Generic Treatments
      • 5.2.1.2 Rilutek: Revenue Forecast, 2013-2024
  • 5.3 Myasthenia Gravis Treatments
    • 5.3.1 Mestinon (Pyridostigmine bromide; Valeant Pharmaceuticals)
      • 5.3.1.1 Lack of Novel Treatments to Compete With Branded Drug
      • 5.3.1.2 Mestinon: Revenue Forecast, 2013-2024
    • 5.3.2 Imuran (Azathioprine; Prometheus Laboratories/Aspen Pharmacare)
      • 5.3.2.1 Serious Adverse Events May Further Reduce Use of Imuran
      • 5.3.2.2 Imuran: Revenue Forecast, 2013-2024

6. Orphan CNS Drugs: R&D Pipelines

  • 6.1 Drugs in the Huntington's Disease Pipeline
    • 6.1.1 ALN-HTT (Alnylam Pharmaceuticals/Medtronic Inc.): Drug-Device Combination Therapy
    • 6.1.2 HD-02 (Avicena Group Inc.): Correcting the Body's Cellular Energy
    • 6.1.3 CERE-120 (Ceregene Inc.): Delivering Neurotrophic Factors to Reverse Disease Process
    • 6.1.4 Lead Compounds (Chaperone Therapeutics): Targeting Protein Misfolding
    • 6.1.5 CX929 (Cortex Pharmaceuticals): Ampakine Treatment to Encourage Neuronal Growth
    • 6.1.6 EPI-743 (Edison Pharmaceuticals): Redox Therapy in Preclinical Testing
    • 6.1.7 GSK356278 (GSK): Respiration Treatment in Line for Additional HD Indication
    • 6.1.8 Modified Antisense Oligo-Nucleotides (Isis Pharmaceuticals): Partnering with Roche to Aid Drug Discovery
    • 6.1.9 NsG33 (Meteorin; NsGene A/S): Neurotrophic Protein Shown to Stimulate Neurite Growth in Preclinical Studies
    • 6.1.10 LSD-1/MAO-B Inhibitor (Oryzon Genomics): Reducing Enzyme Activity May Provide Therapeutic Benefit
    • 6.1.11 PBT2 (Prana Biotechnology): Targeting Metal-Protein Interactions to Reduce Protein Aggregation
    • 6.1.12 PRO289 (Prosensa): Lowering Levels of Mutant Gene Proteins
    • 6.1.13 RP103 (Raptor Pharmaceutical Corporation): Cysteamine Treatment Receives Orphan Designation
    • 6.1.14 Zinc DNA Transcription Factors (Sangamo Biosciences/Shire AG): Switching Genes On and Off
    • 6.1.15 Selisistat (SEN196; Siena Biotechnology): Orphan Drug Targeting Sirtuin Protein Inhibition in Phase II Development
    • 6.1.16 Huntexil (Teva Pharmaceuticals): Neutralizing Dopaminergic Activity
  • 6.2 Drugs in the Amyotrophic Lateral Sclerosis Pipeline, 2013
    • 6.2.1 ALS-02, ALS-05 and ALS-08 (The Avicena Group): Designated Orphan Drug in Advanced Clinical Testing
    • 6.2.2 NurOwn™ (BrainStorm Cell Therapeutics): Stem Cell Treatment to Replace Damaged Cells
    • 6.2.3 Tirasemtiv (CK-2017357; Cytokinetics): Skeletal Muscle Activator Advances to Phase II Trials
    • 6.2.4 E0302 (Mecobalamin; Eisai): Vitamin B12 Therapy Shown to Improve Muscle Wasting
    • 6.2.5 Ozanezumab (GSK1223249; GSK): Monoclonal Antibody Treatment Recruiting Patients for Phase II Trial
    • 6.2.6 Radicut (Edaravone; Mitsubishi Tanabe): Free Radical Scavenger in Advanced Trials
    • 6.2.7 Region Specific Stem Cells (Neuralstem): Neuronal Stem Cell Therapy in Preclinical Testing
    • 6.2.8 NP001 (Neuraltus Pharmaceuticals): Altering Macrophage Activity
    • 6.2.9 sNN0029 (NeuroNova): Growth Factor Treatment Shown to Improve Muscle Strength
    • 6.2.10 TDI-132 (Gilenya™; Novartis & The ALS Therapy Development Institute): Therapy to Counteract Aggressive Immune Cells
  • 6.3 Drugs in the Myasthenia Gravis Pipeline, 2013
    • 6.3.1 Soliris® (Eculizumab; Alexion Pharmaceuticals): Blood Disorder Treatment Investigated as Potential MG Therapy
    • 6.3.2 BHT-3034 (Bayhill Therapeutics): Vaccine Therapy to Reverse Harmful Immune Responses
    • 6.3.3 BKT130 (Biokine Therapeutics): Chemokine Inhibitor
    • 6.3.4 CK-2017357 (Tirasemtiv; Cytokinetics): Designated Orphan Drug Tested in Multiple Indications

7. Orphan Drugs in CNS: Industry Trends, 2014-2024

  • 7.1 Orphan Drugs in CNS: Strengths and Weaknesses, 2013-2024
  • 7.2 Orphan Drugs in CNS: Opportunities and Threats, 2013-2024
  • 7.3 Orphan Drugs in CNS: STEP Analysis, 2013-2024
    • 7.3.1 Social Factors
      • 7.3.1.1 Ethical Considerations in Funding Orphan Drug Development
    • 7.3.2 Technological Developments
      • 7.3.2.1 Increased Understanding of Disease Will Drive Innovation
    • 7.3.3 Economic Pressures
      • 7.3.3.1 Discounts in Drug Prices Will Increase Accessibility
    • 7.3.4 Political Issues
      • 7.3.4.1 Harmonising Global Regulatory Pathways
  • 7.4 Trends in Orphan CNS Drug Development
    • 7.4.1 Clinical Trials for Orphan Drugs
      • 7.4.1.1 Are Adverse Events More Acceptable for Orphan Drugs?
      • 7.4.1.2 Challenges and Opportunities in Patient Recruitment
    • 7.4.2 Biomarkers for Orphan CNS Indications
      • 7.4.2.1 Companion Diagnostics: Driving Personalised Medicine
    • 7.4.3 The Role of Public Funding in Orphan Drug Development
  • 7.5 Commercialising Orphan Drugs in CNS
    • 7.5.1 Pricing of Orphan Drugs May Become Crucial to Success
    • 7.5.2 Market and Patient Access Are Crucial for Orphan Drug Success
    • 7.5.3 Repositioning Drugs for Orphan CNS Indications
      • 7.5.3.1 Expanding Beyond Orphan Indications
    • 7.5.4 Big Pharma and Orphan Drugs
      • 7.5.4.1 Orphan Drugs Fit the Personalised Medicine Model
    • 7.5.5 Common Market Entry Strategies
    • 7.5.6 Orphan Indications as an Opportunity for Generic Drug Manufacturers

8. Research Interviews

  • 8.1 Dr Tsao-Nivaggioli, CEO and Board Member, Avicena Group
    • 8.1.1 Avicena Group
    • 8.1.2 Rare Diseases and Applying for Orphan Drug Status
    • 8.1.3 HD-02: Targeting the Body's Cellular Energy Systems
    • 8.1.4 Challenges in Developing Orphan Drugs
    • 8.1.5 Future Growth Opportunities and Perspective for the Market
  • 8.2 Respondent from Edison Pharmaceuticals
    • 8.2.1 Edison Pharmaceuticals
    • 8.2.2 Defining Orphan Disease and Drug Submissions in Target Markets
    • 8.2.3 The Market Potential (and Risk) of Pursuing Treatments for Mitochondrial Diseases
    • 8.2.4 Company Alliances and Future Prospects for the Market

9. Conclusions from Our Study

  • 9.1 The State of the Orphan CNS Drugs Market
  • 9.2 Renewed Interest in Rare CNS Diseases will Stimulate that Market
  • 9.3 The US will Maintain its Market Dominance from 2014-2024
  • 9.4 High Price of Treatments May Limit Market Potential

List of Tables

  • Table 1.1 Currency Exchange Rates
  • Table 1.2 Common Abbreviations Used in this Report
  • Table 2.1 EU Prevalence Statistics for Selected Rare CNS Indications, 2013
  • Table 3.1 Global Orphan CNS Drug Market: Revenue ($m) and Market Share (%) by Sector, 2012
  • Table 3.2 Global Orphan CNS Drug Market: Overall Market and Revenue Forecasts by Sector ($m, AGR %, CAGR %), 2013-2024
  • Table 3.3 Global Orphan CNS Drug Market: Submarket Shares (%), 2012-2024
  • Table 3.4 Huntington's Disease Submarket: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 3.5 Amyotrophic Lateral Sclerosis Submarket: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 3.6 Myasthenia Gravis Submarket: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.1 Global Orphan CNS Drug Market: Revenue ($m) and Market Share (%) by Region, 2012
  • Table 4.2 Global Orphan CNS Drug Market: Revenue Forecasts ($m, AGR %, CAGR %) by Region, 2013-2024
  • Table 4.3 Global Orphan CNS Drug Market: Regional Submarket Shares (%),2012-2024
  • Table 4.4 US Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.5 EU5 Orphan CNS Drug Market: Revenue ($m) and Market Share (%) by Country, 2012
  • Table 4.6 EU5 Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %) by Country, 2013-2024
  • Table 4.7 Fee Reductions (%) Available for SMEs Developing Orphan Drugs in the EU, 2013
  • Table 4.8 German Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.9 French Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.10 UK Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.11 Spanish Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.12 Italian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.13 Japanese Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.14 Chinese Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.15 Indian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.16 Russian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 4.17 Brazilian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 5.1 Top Orphan CNS Drugs: Revenues ($m) and Leading Revenue Shares (%), 2012
  • Table 5.2 Xenazine: Revenue ($m), 2009-2012
  • Table 5.3 Xenazine: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 5.4 Rilutek: Revenue ($m), 2010-2012
  • Table 5.5 Rilutek: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 5.6 Mestinon: Revenue ($m), 2010-2012
  • Table 5.7 Mestinon: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 5.8 Imuran: Revenue ($m), 2008-2012
  • Table 5.9 Imuran: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024
  • Table 6.1 Selected Pipeline Candidates in Development for Huntington's Disease, 2012
  • Table 6.2 Selected Pipeline Candidates in Development for ALS, 2013
  • Table 6.3 Selected Pipeline Candidates in Development for Myasthenia Gravis, 2013
  • Table 7.1 Global Orphan CNS Drug Market: Strengths and Weaknesses, 2013-2024
  • Table 7.2 Global Orphan CNS Drug Market: Opportunities and Threats, 2013-2024
  • Table 9.1 Global Orphan CNS Drug Market: Revenue ($m, CAGR %), and Market Share (%) by Sector, 2012, 2018 & 2024
  • Table 9.2 Global Orphan CNS Drug Market: Revenue ($m, CAGR %) and Market Share (%) by Region, 2012, 2018 & 2024

List of Figures

  • Figure 2.1 Orphan Disease Prevalence by Country/Region, 2013
  • Figure 2.2 Orphan Drugs Clinical Pipeline by Therapeutic Category, 2012
  • Figure 2.3 Orphan Drug Designation Benefits in Major Markets, 2013
  • Figure 3.1 Orphan Drug Approvals in the US, 2000-2013
  • Figure 3.2 Global Orphan CNS Drug Market: Share (%) by Sector, 2012
  • Figure 3.3 Global Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 3.4 Global Orphan CNS Drug Market: Submarket Shares (%), 2018
  • Figure 3.5 Global Orphan CNS Drug Market: Submarket Shares (%), 2024
  • Figure 3.6 Global Orphan CNS Drug Market: Drivers and Restraints, 2014-2024
  • Figure 3.7 Huntington's Disease Market: Revenue Forecast ($m), 2013-2024
  • Figure 3.8 Amyotrophic Lateral Sclerosis Market: Revenue Forecast ($m), 2013-2024
  • Figure 3.9 Myasthenia Gravis Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.1 Global Orphan CNS Drug Market: Share (%) by Region, 2012
  • Figure 4.2 Global Orphan CNS Drug Market: Share (%) by Region, 2018
  • Figure 4.3 Global Orphan CNS Drug Market: Share (%) by Region, 2024
  • Figure 4.4 US Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.5 EU5 Orphan CNS Drug Market: Share (%) by Country, 2012
  • Figure 4.6 EU5 Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.7 German Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.8 French Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.9 UK Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.10 Spanish Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.11 Italian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.12 Japanese Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.13 Chinese Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.14 Indian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.15 Russian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 4.16 Brazilian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024
  • Figure 5.1 Top Orphan CNS Drug: Revenue Shares (%), 2012
  • Figure 5.2 Xenazine: Revenue ($m), 2009-2012
  • Figure 5.3 Xenazine: Revenue Share (%) by Region, 2012
  • Figure 5.4 Xenazine: Revenue Forecast ($m), 2013-2024
  • Figure 5.5 Rilutek: Revenue ($m), 2010-2012
  • Figure 5.6 Rilutek: Revenue Forecast ($m), 2013-2024
  • Figure 5.7 Mestinon: Revenue ($m), 2010-2012
  • Figure 5.8 Mestinon: Revenue Forecast ($m), 2013-2024
  • Figure 5.9 Imuran: Revenue ($m), 2008-2012
  • Figure 5.10 Imuran: Revenue Forecast ($m), 2013-2024
  • Figure 9.1 Global Orphan CNS Drug Market: Revenue ($m) by Sector, 2012, 2018 & 2024
  • Figure 9.2 Global Orphan CNS Drug Market: Revenue ($m) by Region, 2012, 2018 & 2024

Companies Listed

  • Alexion Pharmaceuticals
  • Agência Nacional de Vigilância Sanitária (ANVISA, Brazil)
  • Alnylam Pharmaceuticals
  • Apotex
  • Aspen Pharmacare
  • Avicena Group
  • Bayhill Therapeutics
  • BHR Pharma
  • Biogen Idec
  • Biokine Therapeutics
  • Biovail
  • BrainStorm Cell Therapeutics
  • California's Stem Cell Agency
  • Cambridge Laboratories
  • Ceregene
  • Chaperone Therapeutics
  • Committee for Orphan Medicinal Products (COMP)
  • Core Pharmaceuticals
  • Cortex Pharmaceuticals
  • Covis Pharmaceuticals
  • Cytokinetics
  • Edison Pharmaceuticals
  • Eisai
  • European Commission
  • European Medicines Agency (EMA)
  • European Union
  • Food and Drug Administration (FDA, US)
  • Glenmark Generics
  • Global Pharmaceuticals (generics division of Impax Laboratories
  • GlaxoSmithKline (GSK)
  • Huntington's Disease Society of America (HDSA)
  • Isis Pharmaceuticals
  • Lundbeck
  • Massachusetts General Hospital
  • Medtronic
  • Ministry of Health, Labor and Welfare (MHLW, Japan)
  • Mitsubishi Tanabe
  • National Institute for Health and Care Excellence (NICE)
  • National Institute for Neurological Disorders and Stroke (NINDS)
  • Neuralstem
  • Neuraltus Pharmaceuticals
  • NeuroNova
  • NeuroSearch
  • Novartis
  • NsGene
  • Omerus Corporation
  • Oryzon Genomics
  • Pfizer
  • Prana Biotechnology
  • Prestwick Pharmaceuticals
  • Prometheus Laboratories
  • Prosensa
  • Raptor Pharmaceuticals
  • Rare Disease UK
  • Roche
  • Roxane Pharmaceuticals
  • Russian Ministry of Health and Social Development (MOHSD)
  • Sangamo Biosciences
  • Sanofi
  • Shire
  • Siena Biotechnology
  • State Food and Drug Administration (SFDA, China)
  • Sun Pharmaceutical Industries
  • Teva Pharmaceuticals
  • The ALS Therapy Development Institute
  • University Hospital Tübingen
  • University of Kyoto
  • Valeant Pharmaceuticals
  • World Health Organisation (WHO)
  • Zydus Pharmaceuticals
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