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OpportunityAnalyzer:全球囊狀纖維化症(CF)治療藥物市場:市場機會分析與未來預測

OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2025

出版商 GlobalData 商品編碼 270218
出版日期 內容資訊 英文 209 Pages
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OpportunityAnalyzer:全球囊狀纖維化症(CF)治療藥物市場:市場機會分析與未來預測 OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2025
出版日期: 2017年04月01日 內容資訊: 英文 209 Pages
簡介

本報告提供囊狀纖維化症(以下CF)治療法研究以及開發的最新趨勢調查,相關的市場預測,疾病概要和目前的治療方法,未滿足需求·市場機會的評估,今後10年的發病數量預測值,臨床試驗的進展,新制治療藥物的開發平台評估,今後的市場趨勢等的調查預測,並將結果概述為以下內容。

第1章 目錄

第2章 簡介

第3章 疾病概要

  • 病因與病情
    • 病因
    • 病情
    • 預測
    • 生活品質(QOL)
  • 徵兆

第4章 免疫學

  • 風險要素和併發症
  • 全球各國到現在為止的趨勢
    • 美國
    • 法國
    • 德國
    • 義大利
    • 西班牙
    • 英國
  • 預測手法
    • 利用的資訊來源
    • 預測的假設·手法
    • 沒利用的資訊來源
  • 免疫學的預測
    • 得病率(整體)
    • 得病率(各年齡)
    • 得病率(男女)
    • 得病率(突變的各類型)
  • 議論
    • 免疫學的趨勢相關結論
    • 分析的限制
    • 分析的優勢

第5章 目前治療選擇

  • 概要
  • 產品簡介:領導品牌,吸入式抗生素
    • TOBI(Tobramycin)
    • TOBI Podhaler(Tobramycin吸入用粉末式)
    • Bramitob (Tobramycin)
    • Colistimethate Sodium(噴霧式:以各種學名藥名流通)
    • Colobreathe(colistimethate·鈉乾燥粉末式)
    • Cayston(Aztreonam)
  • 產品簡介:領導品牌,粘液溶解藥
    • Pulmozyme(dornase alfa)
    • Bronchitol(mannitol)
  • 產品簡介:領導品牌,CFTR(囊狀纖維化症膜電導控制因素)調製器
    • Kalydeco (ivacaftor, VX-770)
  • 產品簡介:領導品牌,其他治療方法
    • 胰臟酵素替代療法(PERTs)

第6章 未滿足需求的評估與機會分析

  • 概要
  • 未滿足需求分析
    • 根治性治療的開發
    • CF相關的肺感染疾病的改善療法
    • 粘液溶解藥的呼吸道沖洗改善
    • 治療方法的遵守相關改善
    • 安全的抗炎治療開發
  • 市場機會分析
    • CFTR蛋白質功能作為標的療法
    • 吸入式抗生素的新層級與處方
    • 新的粘液溶解藥
    • 新的抗發炎醫藥品

第7章 研究開發(R&D)策略

  • 概要
    • 最配合策略
    • 個人化醫療的方法
    • 想法實現的各種研究
    • 許可證給予與產業合作
  • 臨床實驗的設計

第8章 開發平台分析

  • 概要
  • 臨床實驗階段的有潛力的藥劑
  • 臨床實驗階段的有潛力的藥劑:吸入式抗生素
    • Aeroquin (levofloxacin, MP-376)
    • Arikace (amikacin) 85
  • 臨床實驗階段的有潛力藥劑:CFTR調製器
    • Lumacaftor (VX-809)/Kalydeco (VX-770)
    • Ataluren (PTC124)
  • 臨床實驗階段的有潛力的藥劑:PERTs
    • Liprotamase (LY3031642)
  • 初期階段的創新方法

第9章 開發平台評估分析

  • 主要的開發平台藥物臨床方面的標準
  • 主要的開發平台藥物的商業方面的標準
  • 競爭力的評估
  • 今後5年的銷售額的預測
    • 美國
    • 歐洲主要5個國家

第10章 附錄

圖表一覽

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目錄

Cystic fibrosis (CF), which has been given a the diagnostic code E84 from The International Classification of Diseases, Tenth Edition (ICD-10), is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for the CFTR protein. The CFTR protein is mainly located at the apical membrane (the surface of the plasma membrane that faces inward to the lumen) of polarized epithelial tissues, but expression of the CFTR gene in the body is widespread. CFTR protein has been found in tissues such as cardiac myocytes, smooth muscle, erythrocytes, and immune cells (Lubamba et al., 2012).

Global (7MM) sales for the cystic fibrosis (CF) market are forecast by GlobalData to grow from $2.1B in 2015 to $7.6B in 2025. The majority of sales are expected to come from the US, which will represent 68% of the market by 2025. The sales growth during the 2015-2025 forecast periods will be primarily driven by the launches of Vertex's cystic fibrosis transmembrane conductance regulator (CFTR) modulator ivacaftor/tezacaftor. The CFTR modulator drug class is expected to grow from 43.3% of total CF drug sales in 2015 to 81.2% of total CF drug sales in 2025. Vertex Pharmaceuticals will continue to dominate the CF drug space as it has two marketed CFTR modulators and is expected to launch third CFTR modulator during the forecast period, fulfilling some of the major unmet needs of the CF population.

Major drivers of the growth of the CF market over the forecast period will include -

  • The introduction of the high-priced, disease-modifying drug Orkambi (ivacaftor + lumacaftor) in 2015 followed by the introduction of novel, high-priced, disease-modifying CFTR modulators during the forecast period.
  • The continued growth of the number of CF patients due to the overall population growth and continuously increasing life expectancy of CF patients.

R&D strategies in the CF space have focused on symptomatic relief and have targeted organ-specific sequelae of the underlying disease. This approach has mainly resulted in the development of new formulations of antibiotics that can be administered via inhalation for the management of chest infections, and therapies that enhance airway mucociliary clearance. However, over the last decade, advanced technologies have yielded a shift in research development towards therapies that target the underlying cause of the disease. These therapies aim to enhance CFTR function by either restoring wild-type CFTR protein expression or modulating the function of mutant CFTR proteins in cells (Mayer-Hamblett et al., 2016).

The report "OpportunityAnalyzer: Cystic Fibrosis - Opportunity Analysis and Forecasts to 2025" provides overview of cystic fibrosis, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

In particular, this report enable you the following -

  • Annualized CF therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2015 and forecast for ten years to 2025.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the CF therapeutics market.
  • Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the global CF therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Companies mentioned in this report: AbbVie, Chiesis USA, Forest Laboratories, Gilead, Insmed Therapeutics, Johnson & Johnson, Novartis, Pharmaxis, Raptor Pharmaceuticals, Roche, Vertex Pharmaceuticals.

Scope

  • Overview of cystic fibrosis, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized CF therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2015 and forecast for ten years to 2025.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the CF therapeutics market.
  • Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the global CF therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

The report will enable you to -

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.
  • Develop business strategies by understanding the trends shaping and driving the global CF therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global CF therapeutics market in future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Track drug sales in the global CF therapeutics market from 2015-2025.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Table of Contents

1 Table of Contents

1 Table of Contents 2

  • 1.1 List of Tables 6
  • 1.2 List of Figures 7

2 Cystic Fibrosis: Executive Summary 9

  • 2.1 CF Transmembrane Conductance Regulator Modulators Expected to Drive Substantial Growth in the Cystic Fibrosis Market from 2015-2025 9
  • 2.2 Licensing Activity and the Formation of New Partnerships Will Steer Small Companies to Continue Research of Novel Compounds 11
  • 2.3 Marketed and Pipeline CF Transmembrane Conductance Regulator Modulators Will Provide Appropriate Treatment for the Majority of the CF Population, but the High Unmet Needs Will Remain 12
  • 2.4 Opportunities Will Remain for the Development of New CF Transmembrane Conductance Regulator Modulators 12
  • 2.5 What Do Physicians Think? 13

3 Introduction 15

  • 3.1 Catalyst 15
  • 3.2 Related Reports 15
  • 3.3 Upcoming Related Reports 15

4 Disease Overview 16

  • 4.1 Etiology and Pathophysiology 16
    • 4.1.1 Etiology 16
    • 4.1.2 Pathophysiology 16
  • 4.2 Classification 20
  • 4.3 Symptoms 21
  • 4.4 Prognosis 22
  • 4.5 Quality of Life 23

5 Epidemiology 24

  • 5.1 Disease Background 24
  • 5.2 Risk Factors and Comorbidities 25
  • 5.3 Global Trends 26
    • 5.3.1 Diagnosed Incidence and Diagnosed Prevalence 27
  • 5.4 Forecast Methodology 29
    • 5.4.1 Sources Used 29
    • 5.4.2 Forecast Assumptions and Methods 37
    • 5.4.3 Sources Not Used 49
  • 5.5 Epidemiological Forecast for CF (2015-2025) 50
    • 5.5.1 Diagnosed Incident Cases, Based on Registry Data 50
    • 5.5.2 Diagnosed Prevalent Cases, Based on Registry Data 53
    • 5.5.3 CF Mortality, Based on Registry Data 62
    • 5.5.4 Diagnosed Incident Cases, Adjusted for Underestimation 65
    • 5.5.5 Diagnosed Prevalent Cases, Adjusted for Underestimation 68
    • 5.5.6 CF Mortality, Adjusted for Underestimation 76
  • 5.6 Discussion 79
    • 5.6.1 Epidemiological Forecast Insight 79
    • 5.6.2 Limitations of the Analysis 80
    • 5.6.3 Strengths of the Analysis 81

6 Current Treatment Options 82

  • 6.1 Overview 82
  • 6.2 Diagnosis and Treatment 83
    • 6.2.1 Diagnosis 83
    • 6.2.2 Treatment Guidelines and Leading Prescribed Drugs 84
  • 6.3 Clinical Practice 85
  • 6.4 Product Profiles - Major Brands, Inhaled Antibiotics 86
    • 6.4.1 TOBI (tobramycin) 86
    • 6.4.2 TOBI Podhaler (tobramycin) 89
    • 6.4.3 Quinsair (levofloxacin) 93
    • 6.4.4 Cayston (aztreonam) 95
    • 6.4.5 Bramitob/Bethkis (tobramycin) 99
    • 6.4.6 Colobreathe (colistimethate sodium) 102
  • 6.5 Product Profiles - Major Brands, Mucolytics 105
    • 6.5.1 Pulmozyme (dornase alfa) 105
    • 6.5.2 Bronchitol (mannitol) 109
  • 6.6 Product Profiles - Major Brands, CF Transmembrane Conductance Regulator Modulators 113
    • 6.6.1 Kalydeco (ivacaftor) 113
    • 6.6.2 Orkambi (ivacaftor + lumacaftor) 118
  • 6.7 Product Profiles - Major Brands, Other Therapies 121
    • 6.7.1 Pancreatic Enzyme Replacement Therapies 121

7 Unmet Needs Assessment and Opportunity Analysis 124

  • 7.1 Overview 124
  • 7.2 Development of More Effective or Curative Therapies 125
    • 7.2.1 Unmet Need 125
    • 7.2.2 Gap Analysis 126
    • 7.2.3 Opportunity 127
  • 7.3 Development of Chronic Lung Infection Therapies 127
    • 7.3.1 Unmet Need 127
    • 7.3.2 Gap Analysis 128
    • 7.3.3 Opportunity 130
  • 7.4 Increased Treatment Compliance 131
    • 7.4.1 Unmet Need 131
    • 7.4.2 Gap Analysis 132
    • 7.4.3 Opportunity 132
  • 7.5 Development of Mucolytic Therapies 133
    • 7.5.1 Unmet Need 133
    • 7.5.2 Gap Analysis 133
    • 7.5.3 Opportunity 134

8 R&D Strategies 135

  • 8.1 Overview 135
    • 8.1.1 Reformulation Strategies 135
    • 8.1.2 Personalized Treatment Approach 136
    • 8.1.3 Development of Agents with Novel Mechanisms of Action 138
    • 8.1.4 Licensing and Alliances 139
  • 8.2 Clinical Trial Design 139
    • 8.2.1 Current Clinical Trials 139
    • 8.2.2 CF Transmembrane Conductance Regulator Modulator Clinical Trials 140

9 Pipeline Assessment 142

  • 9.1 Overview 142
  • 9.2 Promising Drugs in Clinical Development 142
  • 9.3 Promising Drugs in Clinical Development, Inhaled Antibiotics 144
    • 9.3.1 Arikace (liposomal amikacin) 144
  • 9.4 Promising Drugs in Clinical Development, CF Transmembrane Conductance Regulator Modulators 148
    • 9.4.1 Ivacaftor/Tezacaftor 148
  • 9.5 Innovative Early-Stage Approaches 153

10 Pipeline Valuation Analysis 158

  • 10.1 Clinical Benchmark of Key Pipeline Drugs 158
  • 10.2 Commercial Benchmark of Key Pipeline Drugs 159
  • 10.3 Competitive Assessment 161
  • 10.4 Top-Line 10-Year Forecast 162
    • 10.4.1 US 167
    • 10.4.2 5EU 169
    • 10.4.3 Canada 171

11 Appendix 174

  • 11.1 Bibliography 174
  • 11.2 Abbreviations 186
  • 11.3 Methodology 190
  • 11.4 Forecasting Methodology 190
    • 11.4.1 Diagnosed CF Patients 190
    • 11.4.2 Percent Drug-Treated Patients 190
    • 11.4.3 Drugs Included in Each Therapeutic Class 191
    • 11.4.4 Launch and Patent Expiry Dates 192
    • 11.4.5 General Pricing Assumptions 193
    • 11.4.6 Drug Assumptions 194
    • 11.4.7 Pricing of Pipeline Agents 196
  • 11.5 Primary Research - Key Opinion Leaders Interviewed for This Report 198
  • 11.6 Primary Research - Prescriber Survey 202
  • 11.7 About the Authors 203
    • 11.7.1 Analyst 203
    • 11.7.2 Reviewer 203
    • 11.7.3 Therapy Area Director 203
    • 11.7.4 Epidemiologist 204
    • 11.7.5 Managing Epidemiologist 205
    • 11.7.6 Global Director of Therapy Analysis and Epidemiology 206
    • 11.7.7 Global Head of Healthcare 207
  • 11.8 About GlobalData 208
  • 11.9 Contact Us 208
  • 11.10 Disclaimer 208

List of Tables

1.1 List of Tables

  • Table 1: Cystic Fibrosis: Key Metrics in the Seven Major Pharmaceutical Markets 9
  • Table 2: Onset of CF by Approximate Age 18
  • Table 3: Classification and Criteria of the CFTR Gene Mutations in CF 20
  • Table 4: Symptoms of CF 22
  • Table 5: Risk Factors and Comorbidities of CF 26
  • Table 6: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of CF (Based on

Registry Data) 30

  • Table 7: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of CF (Based on Registry Data) 31
  • Table 8: 7MM, Sources Used for Segmentation of Diagnosed Prevalent Cases of CF by Specific Mutations (Based on Registry Data) 32
  • Table 9: 7MM, Sources Used to Forecast the Proportion of Diagnosed Prevalent Cases of CF by Chronic MRSA Infection (Based on Registry Data) 35
  • Table 10: 7MM, Sources Used to Forecast CF Deaths (Based on Registry Data) 36
  • Table 11: 7MM, Proportion of CF Cases Reported to Respective CF Registries (Primary Research) 36
  • Table 12: 7MM, Sources Not Used in the Epidemiological Analysis of CF 49
  • Table 13: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data) 50
  • Table 14: 7MM, Sex-Specific Diagnosed Incident Cases of CF, All Ages, N (Row %), 2015 (Based on Registry Data) 52
  • Table 15: 7MM, Diagnosed Prevalent Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data) 54
  • Table 16: 7MM, Age-Specific Diagnosed Prevalent Cases of CF, Both Sexes, N (Row %), 2015 (Based on Registry Data) 55
  • Table 17: 7MM, Sex-Specific Diagnosed Prevalent Cases of CF, All Ages, N (Row %), 2015 (Based on Registry Data) 57
  • Table 18: 7MM, Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data) 61
  • Table 19: 7MM, CF Deaths, All Ages, Both Sexes, N, Selected Years 2015-2025 (Based on Registry Data) 62
  • Table 20: 7MM, Sex-Specific CF Deaths, All Ages, N (Row %), 2015 (Based on Registry Data) 64
  • Table 21: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation) 66
  • Table 22: 7MM, Sex-Specific Diagnosed Incident Cases of CF, All Ages, N (Row %), 2015 (Adjusted for Underestimation) 67
  • Table 23: 7MM, Diagnosed Prevalent Cases of CF, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation) 69
  • Table 24: 7MM, Age-Specific Diagnosed Prevalent Cases of CF, Both Sexes, N (Row %), 2015 (Adjusted for Underestimation) 70
  • Table 25: 7MM, Sex-Specific Diagnosed Prevalent Cases of CF, All Ages, N (Row %), 2015 (Adjusted for Underestimation) 72
  • Table 26: 7MM, Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation) 75
  • Table 27: 7MM, CF Deaths, All Ages, Both Sexes, N, Selected Years 2015-2025 (Adjusted for Underestimation) 76
  • Table 28: 7MM, Sex-Specific CF Deaths, All Ages, N (Row %), 2015 (Adjusted for Underestimation) 78
  • Table 29: Treatment Guidelines for CF 84
  • Table 30: Leading Treatments for CF, 2016 85
  • Table 31: Product Profile - TOBI 87
  • Table 32: TOBI SWOT Analysis, 2016 88
  • Table 33: Product Profile - TOBI Podhaler 90
  • Table 34: TOBI Podhaler SWOT Analysis, 2016 92
  • Table 35: Product Profile - Quinsair 94
  • Table 36: Quinsair SWOT Analysis, 2016 95
  • Table 37: Product Profile - Cayston 96
  • Table 38: Cayston SWOT Analysis, 2016 99
  • Table 39: Product Profile - Bramitob 100
  • Table 40: Bramitob SWOT Analysis, 2016 102
  • Table 41: Product Profile - Colobreathe 103
  • Table 42: Colobreathe SWOT Analysis, 2016 105
  • Table 43: Product Profile - Pulmozyme 107
  • Table 44: Pulmozyme SWOT Analysis, 2016 109
  • Table 45: Product Profile - Bronchitol 111
  • Table 46: Bronchitol SWOT Analysis, 2016 113
  • Table 47: Product Profile - Kalydeco 115
  • Table 48: Kalydeco SWOT Analysis, 2016 118
  • Table 49: Product Profile - Orkambi 119
  • Table 50: Orkambi SWOT Analysis, 2016 121
  • Table 51: Pancreatic Enzyme Replacement Therapies (PERTs) SWOT Analysis, 2016 123
  • Table 52: Unmet Need and Opportunity in CF, 2016 125
  • Table 53: Key Late-Stage Pipeline Agents for CF, 2016 143
  • Table 54: Comparison of Therapeutic Classes in Development for CF, 2015-2025 143
  • Table 55: Product Profile - Arikace 145
  • Table 56: Insmed's Arikace - Phase III Clinical Trials 146
  • Table 57: Arikace SWOT Analysis, 2016 148
  • Table 58: Product Profile - ivacaftor/tezacaftor 149
  • Table 59: Vertex's ivacaftor/tezacaftor - Phase III Clinical Trials 150
  • Table 60: Efficacy of Ivacaftor + Tezacaftor 151
  • Table 61: Ivacaftor + Tezacaftor SWOT Analysis, 2016 153
  • Table 62: Drugs in Development for CF, 2016 156
  • Table 63: Clinical Benchmark of Key Pipeline Drugs - Inhaled Antibiotics 159
  • Table 64: Clinical Benchmark of Key Pipeline Drugs - CFTR Modulators 159
  • Table 65: Commercial Benchmark of Key Pipeline Drugs - Inhaled Antibiotics 160
  • Table 66: Commercial Benchmark of Key Pipeline Drugs - CFTR Modulators 160
  • Table 67: Top-Line Sales Forecasts ($m) for CF, 2015-2025 163
  • Table 68: Key Events Impacting Sales for CF, 2015-2025 166
  • Table 69: CF Market - Global Drivers and Barriers, 2015?2025 167
  • Table 70: Key Historical and Projected Launch Dates 192
  • Table 71: Key Historical and Projected Patent Expiry Dates 193
  • Table 72: High-Prescribing Physicians (Non-KOLs) Surveyed, By Country 202

List of Figures

1.2 List of Figures

  • Figure 1: Global Sales for CF by Country/Region, 2015 and 2025 11
  • Figure 2: Classes of CFTR Gene Mutations in CF 21
  • Figure 3: 7MM, Diagnosed Incident Cases of CF, All Ages, Both Sexes, N, 2015-2025 (Based on Registry Data) 51
  • Figure 4:
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