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Opportunity Analyzer: Graft-Versus-Host Disease - Opportunity Analysis and Forecasts to 2023

Summary

The main drivers of growth in graft versus host disease over the 2013-2023 forecast period include the increasing numbers of allogeneic hematopoietic stem cell transplants and the increasing use of biologic off-label and marketed therapies in the 6MM.

Sanofi is a key player in the GVHD market with Thymoglobuline and Lemtrada by infiltrating prophylaxis, aGVHD and cGVHD. J&J’s Remicade, has gained a significant patent share of the aGVHD patient group, as it is one of the few drugs that are efficient in GVHD with GI involvement. Gaining more and more ground in the treatment management of cGVHD is Roche’s Rituxan that GlobalData estimates will be one of the bestselling biologics in GVHD in the US by 2023.

Despite the vast array of available off-label therapies for the treatment and/or prevention of GVHD, very few of these therapies have been tested in large randomized trials. This results in vague treatment recommendations and many patients being enrolled in institutional clinical trials. The only available standard of care is intravenous methylprednisolone that fails to produce a complete response in more than 50% of treated patients. Steroid-refractory subgroups face low prognosis with deteriorating quality of life. Second and third life treatments can vary from country to country but also between different medical institutions in the same country.

The GVHD market was valued at $297.0m across the 6MM in 2013, and is expected to increase to $544.4m in 2023, at a Compound Annual Growth Rate (CAGR) of 12.84%.

Highlights

Key Questions Answered

- How will the GVHD market landscape change within the 2013-2023 forecast period in the 6MM?
- What are the most promising late-stage pipeline drugs in GVHD?
- How do the clinical and commercial attributes of late-stage pipeline therapies compare to one another and against existing treatment options?
- What are the unmet needs in GVHD treatment management?
- What needs to change in the GVHD R&D and treatment guidelines settings?

Key Findings

- High uptake of off-label biologics (Rituxan, Lemtrada, Remicade and Thymoglobuline)
- No national guidelines are in place
- GVHD remains a field of highly unattained unmet need
- No consensus over clinical trial endpoints
- Regulatory T cells (Tregs) are the focus of the early stage pipeline
- Huge opportunity exists in the chronic patient segment of GVHD
- Institutionally funded studies are the core of GVHD treatment management

Scope

- Overview of GVHD, including epidemiology, etiology, pathophysiology, symptoms and current treatment options
- Annualized GVHD therapeutics market revenue, annual cost of therapies and forecasts for five years to 2023.
- Key topics covered include strategic product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the GVHD therapeutics market.
- Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanisms of action under development, including TNF blockers, CD25a blockers, GVHD-specific therapies etc.
- Analysis of the current and future market competition in the US and five major EU GVHD therapeutics market. Clinical and commercial benchmarking of promising pipeline products versus standard of care treatments and competitive assessment of all therapies. Insightful review of the key industry drivers, restraints and challenges.

Reasons to buy

The report will enable you to -
- Identify the unmet needs and remaining opportunities in the GVHD therapeutics market.
- Develop business strategies by understanding the trends shaping and driving the US and five major EU GVHD therapeutics market.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Assess the clinical and commercial viability of promising pipeline products.
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
- Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU GVHD therapeutics market in future.

Table Of Contents

Opportunity Analyzer: Graft-Versus-Host Disease - Opportunity Analysis and Forecasts to 2023
1 Table of Contents
1 Table of Contents 11
1.1 List of Tables 17
1.2 List of Figures 20
2 Introduction 22
2.1 Catalyst 22
2.2 Related Reports 23
2.3 Upcoming Related Reports 23
3 Disease Overview 24
3.1 Etiology and Pathophysiology 24
3.1.1 Etiology 24
3.1.2 Pathophysiology 26
3.1.3 Classification and Prognosis 27
3.2 Symptoms 29
4 Epidemiology 31
4.1 Disease Background 31
4.2 Risk Factors and Comorbidities 32
4.3 Global Trends 34
4.4 Forecast Methodology 35
4.4.1 Sources Used 37
4.4.2 Sources Not Used 43
4.4.3 Diagnosed Incident Cases of HSCTs 44
4.4.4 Diagnosed Incident Cases of Autologous HSCTs 45
4.4.5 Diagnosed Incident Cases of Allogeneic HSCTs 46
4.4.6 Diagnosed Incident Cases of aGVHD 48
4.4.7 Four-Year Diagnosed Prevalent Cases of aGVHD 48
4.4.8 Diagnosed Incident Cases of cGVHD 49
4.4.9 Five-Year Diagnosed Prevalent Cases of cGVHD 49
4.5 Epidemiological Forecast for GVHD (2013-2023) 50
4.5.1 Diagnosed Incident Cases of HSCTs 50
4.5.2 Diagnosed Incident Cases of Autologous HSCTs 52
4.5.3 Diagnosed Incident Cases of Allogeneic HSCTs 53
4.5.4 Diagnosed Incident Cases of aGVHD 55
4.5.5 Four-Year Diagnosed Prevalent Cases of aGVHD 57
4.5.6 Diagnosed Incident Cases of cGVHD 58
4.5.7 Five-Year Diagnosed Prevalent Cases of cGVHD 60
4.6 Discussion 62
4.6.1 Epidemiological Forecast Insight 62
4.6.2 Limitations of the Analysis 63
4.6.3 Strengths of the Analysis 64
5 Current Treatment Options 65
5.1 Overview 65
5.2 Product Profiles - Major Therapies 67
5.2.1 Methylprednisolone (numerous brand and generic names) 67
5.2.2 Cyclosporine (numerous brands and generic names) 72
5.2.3 Anti-Thymocyte Globulin (ATG) Therapies 77
5.3 Product Profiles - Off-Label Therapies 82
5.3.1 Biologics (Tumor Necrosis Factor (TNF)? Inhibitors, Interleukin-2 Receptor ? (CD25) Inhibitors, Co-Stimulatory Blockers and Other Biologics) 82
5.3.2 Small Immunosuppressive Molecules (mTOR Inhibitors, Solid Organ Transplant Therapies, and Anti-Neoplastic Therapies) 102
6 Unmet Need and Opportunity 118
6.1 Overview 118
6.2 Lack of Randomized Clinical Trials 121
6.2.1 Unmet Need 121
6.2.2 Gap Analysis 122
6.2.3 Opportunity 122
6.3 No Consensus Regarding Clinical Trial Endpoints 122
6.3.1 Unmet Need 122
6.3.2 Gap Analysis 123
6.3.3 Opportunity 123
6.4 Optimization of Prophylactic Measures 124
6.4.1 Unmet Need 124
6.4.2 Gap Analysis 124
6.4.3 Opportunity 125
6.5 Lack of Standardized Protocols for Established and Off-Label Therapies 125
6.5.1 Unmet Need 125
6.5.2 Gap Analysis 126
6.5.3 Opportunity 126
6.6 Improved Treatment Outcomes for Patients Who Develop Toxicities 127
6.6.1 Unmet Need 127
6.6.2 Gap Analysis 128
6.6.3 Opportunity 128
6.7 Desire for Treatments that Exhibit Longer-Lasting Efficacy Profiles 128
6.7.1 Unmet Need 128
6.7.2 Gap Analysis 129
6.7.3 Opportunity 130
6.8 Management of SR-aGVHD Patients 130
6.8.1 Unmet Need 130
6.8.2 Gap Analysis 131
6.8.3 Opportunity 132
6.9 Management of Sclerotic cGVHD Patients 132
6.9.1 Unmet Need 132
6.9.2 Gap Analysis 133
6.9.3 Opportunity 133
6.10 Improved Prognosis for Patients with Lung Involvement 134
6.10.1 Unmet Need 134
6.10.2 Gap Analysis 134
6.10.3 Opportunity 135
7 Research and Development Strategies 136
7.1 Overview 136
7.1.1 Improvement of Prophylactic Regimens 138
7.1.2 Inhibition of T-Cell Proliferation and Activation 139
7.1.3 Adopting Treatments from Hematologic Indications 140
7.1.4 Targeting Niche Subgroups Within GVHD 140
7.2 Current Clinical Trial Design 142
7.2.1 Current Trial Designs are Dependent on the Stage Within the Treatment Algorithm That a Product is Targeting 142
7.2.2 Lack of Consensus on Clinical Endpoints in Current Trial Designs 142
7.2.3 Patient Exclusion Issues in Current Trial Designs 143
7.2.4 Current Trial Design of Key Pipeline Products 143
7.3 Future Clinical Trial Design 145
7.3.1 Future Trial Designs Need to Incorporate Randomization 145
7.3.2 Future Trial Designs Need to Incorporate Conditioning Regimens and Hematological Patients 146
7.3.3 Companies Behind Off-Label Therapies Should Strategize to Conduct Randomized Studies 146
7.3.4 Companies with Pipeline Assets Targeting GVHD must invest in Biomarkers 146
7.3.5 Design of Early-Phase Clinical Trials for Cellular and Gene Therapy Products Accommodates 2014 Guidance from the FDA 147
8 Pipeline Assessment 148
8.1 Overview 148
8.2 Promising Drugs in Clinical Development 149
8.2.1 Leukotac (inolimomab) 149
8.2.2 Begedina (BT 5/9) 154
8.2.3 Budenofalk (budesonide) 157
8.2.4 ATG-Fresenius (EZ-2053) 160
8.2.5 Prochymal (remestemcel-L) 164
8.2.6 MultiStem (modified mesenchymal stem cells) 169
8.2.7 Uvadex (Extracorporeal Photophoresis) 172
8.2.8 orBec (beclomethasone dipropionate) 176
8.3 Innovative Early-Stage Approaches 179
8.3.1 Targeting Regulatory T Cells 182
8.3.2 Mesenchymal Stem Cell Therapies 183
8.3.3 Immunomodulatory Cell Surface Receptor Inhibitors 184
8.3.4 IL-6 Inhibitors 185
9 Pipeline and Off-Label Valuation Analysis 186
9.1 Clinical Benchmarking of Key Pipeline and Off-Label Drugs 186
9.1.1 GVHD Prophylaxis 186
9.1.2 Acute GVHD 188
9.1.3 Chronic GVHD 193
9.2 Commercial Benchmarking of Key Pipeline and Off-Label Drugs 194
9.2.1 GVHD Prophylaxis 195
9.2.2 Acute GVHD 197
9.2.3 Chronic GVHD 199
9.3 Competitive Assessment 200
9.3.1 GVHD Prophylaxis 200
9.3.2 Acute GVHD 201
9.3.3 Chronic GVHD 203
9.4 Top-Line Ten-Year forecast 205
9.4.1 US 210
9.4.2 5EU 215
10 Appendix 218
10.1 Bibliography 218
10.2 Abbreviations 237
10.3 Methodology 243
10.4 Forecasting Methodology 243
10.4.1 Diagnosed GVHD Patients 243
10.4.2 Percent Drug-Treated Patients 244
10.4.3 Drugs Included in Each Therapeutic Class 244
10.4.4 Launch and Patent Expiry Dates 245
10.4.5 General Pricing Assumptions 245
10.4.6 Individual Drug Assumptions 246
10.4.7 Generic Erosion 253
10.4.8 Pricing of Pipeline Agents 253
10.5 Physicians and Specialists Included in This Study 254
10.6 About the Authors 257
10.6.1 Author 257
10.6.2 Reviewer 257
10.6.3 Epidemiologist 258
10.6.4 Global Head of Healthcare 258
10.7 About GlobalData 259
10.8 Disclaimer 259

1.1 List of Tables
Table 1: Steps Involved in the Development of aGVHD 26
Table 2: Steps Involved in the Development of cGVHD 27
Table 3: Grading System of Organ Involvement in aGVHD 28
Table 4: Classification of Organ Involvement in cGVHD 29
Table 5: Symptoms of aGVHD 29
Table 6: Symptoms of cGVHD 30
Table 7: Risk Factors and Comorbidities for GVHD 33
Table 8: 6MM, Sources of HSCT, aGVHD, and cGVHD Data Used for the Forecast 37
Table 9: 6MM, Sources Not Used in the Epidemiological Analysis of GVHD 44
Table 10: 6MM, Diagnosed Incident Cases of HSCTs, Both Sexes, All Ages, N, 2013-2023 51
Table 11: 6MM, Diagnosed Incident Cases of Autologous HSCTs, Both Sexes, All Ages, N, 2013-2023 52
Table 12: 6MM, Diagnosed Incident Cases of Allogeneic HSCTs, Both Sexes, All Ages, N, 2013-2023 54
Table 13: 6MM, Diagnosed Incident Cases of aGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, Both Sexes, All Ages, N, 2013-2023 56
Table 14: 6MM, Four-Year Diagnosed Prevalent Cases of aGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, All Ages, Both Sexes, N, 2013-2023 57
Table 15: 6MM, Diagnosed Incident Cases of cGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, All Ages, Both Sexes, N, 2013-2023 59
Table 16: 6MM, Five-Year Diagnosed Prevalent Cases of cGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, All Ages, Both Sexes, N, 2013-2023 61
Table 17: Product Profile - Methylprednisolone 68
Table 18: Efficacy of Methylprednisolone in GVHD Studies 70
Table 19: SWOT analysis - Methylprednisolone, 2014 72
Table 20: Product Profile - Cyclosporine 74
Table 21: Efficacy of Cyclosporine in GVHD Studies 75
Table 22: SWOT Analysis - Cyclosporine, 2014 76
Table 23: Product Profiles - ATG Therapies 79
Table 24: Efficacy of ATG Therapies in GVHD 80
Table 25: SWOT Analysis - ATG Therapies, 2014 81
Table 26: Product Profile - Biologics - TNF? Inhibitors 84
Table 27: Efficacy of Anti-TNF? Therapies in GVHD 85
Table 28: Product Profiles - Biologics - IL-2R? (CD25) Inhibitors 88
Table 29: Efficacy of IL-2R? (CD25) Inhibitor Therapies in GVHD 89
Table 30: Product Profile - Biologics - Co-Stimulatory Blockers 92
Table 31: Efficacy of Co-Stimulatory Blocker Therapies in GVHD 93
Table 32: Product Profile - Other Biologics (Campath/Lemtrada and Rituxan) 96
Table 33: Efficacy of Other Biologic Therapies (Campath/Lemtrada and Rituxan) in GVHD 97
Table 34: Safety Profile - Off-Label Biologics Used in GVHD, 2014 99
Table 35: SWOT Analysis - Biologics - TNF? Inhibitors, 2014 100
Table 36: SWOT Analysis - Biologics - IL-2R? (CD25) inhibitors, 2014 101
Table 37: SWOT Analysis - Biologics - Co-Stimulatory Blockers, 2014 101
Table 38: SWOT Analysis - Biologics - Others, 2014 102
Table 39: Product Profile - Small Immunosuppressive Molecules - mTOR Inhibitors 104
Table 40: Efficacy of mTOR Inhibitors in GVHD 105
Table 41: Product Profile - Small Immunosuppressive Molecules - SOT Therapies 106
Table 42: Efficacy of SOT Therapies in GVHD 107
Table 43: Product Profile - Small Immunosuppressive Molecules - Anti-Neoplastic Therapies 110
Table 44: Efficacy of Anti-Neoplastic Therapies in GVHD 112
Table 45: Safety Profile - Small Immunosuppressive Molecules 114
Table 46: SWOT Analysis - Small Immunosuppressive Molecules - mTOR Inhibitors, 2014 115
Table 47: SWOT Analysis - Small Immunosuppressive Molecules - SOT Therapies, 2014 116
Table 48: SWOT analysis - Small Immunosuppressive Molecules - Anti-Neoplastic Therapies, 2014 117
Table 49: Unmet Need and Opportunity in GVHD 120
Table 50: Clinical Trial Design of Key Pipeline Drugs for GVHD, July 2014 144
Table 51: GVHD - Late Stage Pipeline, 2013 149
Table 52: Product Profile - Leukotac (inolimomab) 151
Table 53: Efficacy of Leukotac in GVHD 153
Table 54: SWOT Analysis - Leukotac (inolimomab), 2014 154
Table 55: Product Profile - Begedina (BT 5/9) 155
Table 56: Efficacy of Begedina in GVHD 156
Table 57: SWOT Analysis - Begedina (BT 5/9), 2015 157
Table 58: Product Profile - Budenofalk (budesonide) 158
Table 59: SWOT Analysis - Budenofalk, 2014 159
Table 60: Product Profile - ATG-Fresenius (EZ-2053) 162
Table 61: Efficacy of ATG-Fresenius in GVHD (EZ-2053) 163
Table 62: SWOT Analysis - ATG-Fresenius, 2014 164
Table 63: Product Profile - Prochymal (remestemcel-L) 167
Table 64: SWOT Analysis - Prochymal (remestemcel-L), 2014 169
Table 65: Product Profile - MultiStem (modified mesenchymal stem cells) 170
Table 66: SWOT Analysis - MultiStem (modified mesenchymal stem cells) 171
Table 67: Product Profile - Uvadex (Extracorporeal Photophoresis) 174
Table 68: Efficacy of Uvadex in GVHD 175
Table 69: SWOT Analysis - Uvadex (Extracorporeal Photophoresis) 176
Table 70: Product Profile - orBec (beclomethasone dipropionate) 178
Table 71: SWOT Analysis - orBec (beclomethasone proprionate), 2014 179
Table 72: Early-Stage Pipeline Products for GVHD, July 2014 181
Table 73: Clinical Benchmarking for GVHD Prophylaxis, 2014 188
Table 74: Clinical Benchmarking for aGVHD, 2014 192
Table 75: Clinical Benchmarking for cGVHD, 2014 194
Table 76: Commercial Benchmarking for GVHD Prophylaxis, 2014 196
Table 77: Commercial Benchmarking for aGVHD, 2014 198
Table 78: Commercial Benchmarking for cGVHD, 2014 199
Table 79: Top-Line Sales Forecasts ($m) for GVHD, 2013-2023 207
Table 80: Key Events Impacting Sales for GVHD, 2013-2023 209
Table 81: Global GVHD Market - Drivers and Barriers, 2013-2023 210
Table 82: Key Launch Dates, GVHD, 2013-2023 245
Table 83: Key Patent Expiries, GVHD, 2013-2023 245

1.2 List of Figures
Figure 1: Schematic of HSCT 25
Figure 2: Schematic Flow Chart of the Derivation of the aGVHD and cGVHD Patient Population in the 6MM 35
Figure 3: 6MM, Diagnosed Incident Cases of HSCTs , Both Sexes, All Ages, N, 2013-2023 51
Figure 4: 6MM, Diagnosed Incident Cases of Autologous HSCTs, Both Sexes, All Ages, N, 2013-2023 53
Figure 5: 6MM, Diagnosed Incident Cases of Allogeneic HSCTs, Both Sexes, All Ages, N, 2013-2023 54
Figure 6: 6MM, Diagnosed Incident Cases of aGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, Both Sexes, All Ages, N, 2013-2023 56
Figure 7: 6MM, Four-Year Diagnosed Prevalent Cases of aGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, All Ages, Both Sexes, 2013-2023 58
Figure 8: 6MM, Diagnosed Incident Cases of cGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, All Ages, Both Sexes, N, 2013-2023 60
Figure 9: 6MM, Five-Year Diagnosed Prevalent Cases of cGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs, All Ages, Both Sexes, N, 2013-2023 61
Figure 10: Trends in aGVHD Management in the 6MM, 2014 66
Figure 11: Trends in cGVHD Management in the 6MM, 2014 67
Figure 12: Schematic Representation of Unmet Needs 119
Figure 13: An Overview of the GVHD Prophylaxis Clinical Trials, 2015 136
Figure 14: An Overview of the aGVHD Clinical Trials, 2015 137
Figure 15: An Overview of the cGVHD Clinical Trials, 2015 137
Figure 16: Competitive Assessment of GVHD Prophylaxis Off-Label Therapies and Late-Stage Pipeline Agents, 2013-2023 201
Figure 17: Competitive Assessment of aGVHD Off-Label Therapies and Late-Stage Pipeline Agents, 2013-2023 203
Figure 18: Competitive Assessment of cGVHD Off-Label Therapies and Late-Stage Pipeline Agents, 2013-2023 205
Figure 19: Global Sales for GVHD by Region and GVHD Indication, 2013-2023 208

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