Global Orphan and Rare Dermatology Drugs Market to 2024 - An Increasingly Competitive Landscape as New Companies Enter the Market with Novel Products, Driving Strong Market Growth

GBI Research
141 Pages - GBI10599
$4,995.00

Summary

Dermatology is a highly diverse therapy area, in terms of severity and clinical presentation, which deals with diseases of the skin, hair and nails. Whilst an orphan disease is a disease that is neglected in terms of research and funding and in the US, a disease is considered to be rare if it affects fewer than 200,000 people.

Many orphan and rare dermatology disorders are associated with significant quality of life impairments, particularly if the disease is insufficiently controlled. In particular, disease visibility can have a profoundly negative impact on patient confidence. However, the current therapeutics market is a highly genericized therapy area and there exists a significant unmet need for more efficacious and safer treatment options that treat the underlying causes of disease as opposed to managing the symptoms.

This report covers all orphan and rare dermatology disorders, but there is a particular focus on six key diseases, systemic sclerosis (scleroderma), alopecia, epidermolysis bullosa, pemphigus vulgaris, vitiligo and cutaneous lupus erythematosus, as these conditions have the largest pipelines within the therapy area.

The premium product market size for orphan and rare dermatology therapeutics was valued at $1.64 billion in 2017 and it is expected to grow at a compound annual growth rate (CAGR) of 20.5% to $6.07 billion in 2024.

Scope

- The orphan and rare dermatology market landscape is expected to change and grow. Novel targets are expected to enter the market and drive market growth with a move towards disease modifying drugs instead of drugs which manage symptoms.
- Overall, there are 262 orphan and rare dermatology products in the pipeline.
- Which molecular targets are most prominent within the pipeline?
- How do the key indications differ in terms of molecule type?
- How does the composition of the pipeline compare with that of the existing market?
- What mechanisms of action are most common for pipeline drugs?
- The premium products market size for orphan and rare dermatology therapeutics is expected to grow, from $1.64 billion in 2017 to $6.07 billion in 2024, at a CAGR of 20.5%.
- Which products will contribute to market growth most, and will any achieve blockbuster status?
- Will the current market leaders retain their dominance over the forecast period, and how is their revenue share of the orphan and rare dermatology market set to change?
- What CAGR will these companies register in the forecast period?
- How many strategic consolidations have been completed in the past decade?
- Which types of assets attract the largest deal values?

Reasons to buy

- Understand the current clinical and commercial landscape by considering disease pathogenesis, diagnosis, prognosis and the treatment options available at each stage of diagnosis for the six key indications.
- Visualize the composition of the orphan and rare dermatology market overall and across each key indication, in terms of dominant molecule types and targets, highlighting the key commercial assets and players.
- Identify trends and developments, in terms of molecule type and molecular target, within the overall orphan and rare dermatology pipeline, as well as for each of the six key indications.
- Understand the growth in patient epidemiology and market revenues for the orphan and rare dermatology market globally, and across the key players and product types.
- Consider market opportunities and potential risks by examining the incentives and challenges of drug development within the orphan market space and the trends in clinical trial duration and size, as well as clinical trial failure rates, by stage of development, molecule type and molecular target.
- Recognize how the evolving treatment landscape will drive market growth to 2024 and understand the contributions that different products, molecular targets and companies will make to this growth.
- Identify commercial opportunities in the orphan and rare dermatology deals landscape by analyzing trends in licensing and co-development deals.

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1 Table of Contents
1 Table of Contents 5
1.1 List of Tables 8
1.2 List of Figures 8
2 Introduction 11
2.1 Therapy Area Overview 11
2.1.1 Overview of the Dermatology Market 11
2.1.2 Overview of Orphan and Rare Diseases 11
2.1.3 Overview of Orphan and Rare Dermatological Diseases 12
2.2 Symptoms 14
2.3 Diagnosis 16
2.3.1 Systemic Sclerosis 17
2.3.2 Alopecia 17
2.3.3 Epidermolysis Bullosa 18
2.3.4 Pemphigus Vulgaris 18
2.3.5 Vitiligo 19
2.3.6 Cutaneous Lupus Erythematosus 19
2.4 Etiology 20
2.5 Pathophysiology 21
2.5.1 Systemic sclerosis 21
2.5.2 Alopecia 22
2.5.3 Epidermolysis Bullosa 23
2.5.4 Pemphigus Vulgaris 23
2.5.5 Vitiligo 23
2.5.6 Cutaneous Lupus Erythematosus 24
2.6 Epidemiology 25
2.6.1 Systemic sclerosis 25
2.6.2 Alopecia 26
2.6.3 Epidermolysis Bullosa 26
2.6.4 Pemphigus Vulgaris 26
2.6.5 Vitiligo 27
2.6.6 Cutaneous Lupus Erythematosus 27
2.7 Co-morbidities and Complications 27
2.7.1 Systemic sclerosis 27
2.7.2 Alopecia 28
2.7.3 Epidermolysis Bullosa 28
2.7.4 Pemphigus Vulgaris 28
2.7.5 Vitiligo 29
2.7.6 Cutaneous Lupus Erythematosus 29
2.8 Prognosis 29
2.8.1 Systemic Sclerosis 29
2.8.2 Alopecia 29
2.8.3 Epidermolysis Bullosa 30
2.8.4 Pemphigus Vulgaris 30
2.8.5 Vitiligo 30
2.8.6 Cutaneous Lupus Erythematosus 31
2.9 Treatment 31
2.9.1 Systemic Sclerosis 32
2.9.2 Alopecia 32
2.9.3 Epidermolysis Bullosa 33
2.9.4 Pemphigus Vulgaris 33
2.9.5 Vitiligo 34
2.9.6 Cutaneous Lupus Erythematosus 34
3 Key Marketed Products 36
3.1 Overview 36
3.2 Premium Drugs 38
3.2.1 Humira (adalimumab) - AbbVie 38
3.2.2 Ilaris (canakinumab) - Novartis 40
3.2.3 Afinitor (everolimus) - Novartis 41
3.2.4 Kineret (anakinra) - Swedish Orphan Biovitrum 43
3.2.5 Arcalyst (rilonacept) - Regeneron Pharmaceuticals 45
3.2.6 Tracleer (bosentan) - Johnson & Johnson 47
3.2.7 Thalomid (thalidomide) - Celgene 49
3.3 Highly Genericized Drugs 53
3.3.1 Medrol (methylprednisolone) - Pfizer 53
3.3.2 Meticorten/Rayos (Prednisone) - Merck & Co./Horizon Pharma 55
4 Pipeline Landscape Assessment 58
4.1 Overview 58
4.2 Pipeline by Stage of Development, Molecule Type and Program Type 59
4.2.1 Overall Pipeline 59
4.2.2 Pipeline by Key Indication 60
4.3 Molecular Targets in the Pipeline 63
4.3.1 Overall Pipeline 63
4.3.2 Pipeline by Key Indication 64
4.4 Clinical Trials Landscape 65
4.4.1 Challenges Associated with Clinical Trials for Orphan and Rare Diseases 66
4.4.2 Incentives for Clinical Trials for Orphan and Rare Diseases 67
4.4.3 Clinical Trial Failure Rates 68
4.4.4 Clinical Trial Duration 69
4.4.5 Clinical Trial Size 70
4.4.6 Cumulative Clinical Trial Size 70
4.5 Assessment of Key Pipeline Products 71
4.5.1 Epidiolex (cannabidiol) - GW Pharmaceuticals 72
4.5.2 Episalvan (birch bark extract) - Amryt Pharma 73
4.5.3 KB-103 - Krystal Biotech 74
4.5.4 Givosiran - Alnylam Pharmaceuticals 75
4.5.5 EB-101 - Abeona Therapeutics 77
4.5.6 OTL-103 - Orchard Therapeutics 78
4.5.7 Siponimod - Novartis 79
4.5.8 FCX-007 - Fibrocell Science 80
5 Multi-scenario Market Forecast to 2024 82
5.1 Market Size 82
5.2 Revenue Forecast by Molecular Target 85
5.2.1 Products Acting on Interleukins 85
5.2.2 Products Acting on Tumor Necrosis Factor-Alpha 86
5.2.3 Products Acting on Tyrosine Protein Kinases 87
5.2.4 Products Acting on Hormones and Hormone Receptors 88
6 Company Analysis and Positioning 90
6.1 Revenue and Market Share Analysis by Company 92
6.1.1 Novartis 98
6.1.2 GW Pharmaceuticals 99
6.1.3 AbbVie 100
6.1.4 Roche 101
6.1.5 Pfizer 101
6.1.6 Amryt Pharma 103
6.1.7 GlaxoSmithKline 104
6.1.8 Celgene 105
6.2 Company Landscape 106
6.3 Marketed and Pipeline Portfolio Analysis 107
7 Strategic Consolidations 109
7.1 Licensing Deals 109
7.1.1 Deals by Region, Year and Value 109
7.1.2 Deals by Key Indication and Value 111
7.1.3 Deals by Stage of Development and Value 112
7.1.4 Deals by Molecule Type, Molecular Target and Value 113
7.1.5 Licensing Deals with Disclosed Values 114
7.2 Co-development Deals 115
7.2.1 Deals by Region, Year and Value 115
7.2.2 Deals by Key Indication and Value 116
7.2.3 Deals by Stage of Development and Value 117
7.2.4 Deals by Molecule Type, Molecular Target and Value 118
7.2.5 Co-development Deals with Disclosed Values 119
8 Appendix 120
8.1 References 120
8.2 Figures of All Pipeline Products 129
8.3 Abbreviations 136
8.4 Disease List 137
8.5 Methodology 137
8.5.1 Coverage 137
8.5.2 Secondary Research 138
8.5.3 Market Size and Revenue Forecasts 138
8.5.4 Pipeline Analysis 138
8.5.5 Competitive Landscape 139
8.6 Contact Us 139
8.7 Disclaimer 139

1.1 List of Tables
Table 1: Orphan and Rare Dermatology Drugs Market, Global, Symptoms of the Key Indications, 2018 15
Table 2: Orphan and Rare Dermatology Drugs Market, Global, Etiology of the Key Indications, 2018 20
Table 3: Orphan and Rare Dermatology Drugs Market, Global, Prognosis for Epidermolysis Bullosa, 2018 30
Table 4: Orphan and Rare Dermatology Drugs Market, Global, Key Marketed Products and Approved Indications, 2018 37
Table 5: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Humira, 2018 38
Table 6: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Ilaris, 2018 40
Table 7: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Afinitor, 2018 42
Table 8: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Kineret, 2018 44
Table 9: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Arcalyst, 2018 46
Table 10: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Tracleer, 2018 48
Table 11: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Thalomid, 2018 50
Table 12: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Methylprednisolone, 2018 54
Table 13: Orphan and Rare Dermatology Drugs Market, Global, Approved Orphan and Rare Dermatology Indications for Meticorten, 2018 56
Table 14: Orphan and Rare Dermatology Drugs Market, US, Approved Orphan and Rare Dermatology Indications for Rayos, 2018 56
Table 15: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue Forecast for Key Products ($m), 2017-2024 84
Table 16: Orphan and Rare Dermatology Drugs Market, Global, Forecast Revenue by Company (%), 2017-2024 93

1.2 List of Figures
Figure 1: Orphan and Rare Dermatology Drugs Market, 7MM, Epidemiology Patterns for Systemic Sclerosis (Scleroderma), 2017-2024 26
Figure 2: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Humira ($bn), 2006-2024 39
Figure 3: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Ilaris ($bn), 2009-2024 41
Figure 4: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Afinitor ($bn), 2008-2024 43
Figure 5: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Kineret ($m), 2008-2024 45
Figure 6: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Arcalyst ($m), 2008-2024 47
Figure 7: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Tracleer ($bn), 2006-2024 49
Figure 8: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Thalomid ($m), 2006-2024 52
Figure 9: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Medrol ($m), 2008-2024 55
Figure 10: Orphan and Rare Dermatology Drugs Market, US, Annual Revenue for Rayos ($bn), 2013-2024 57
Figure 11: Orphan and Rare Dermatology Drugs Market, Global, Overall Pharmaceutical Industry Pipeline by Therapy Area, 2018 58
Figure 12: Orphan and Rare Dermatology Drugs Market, Global, Overall Pipeline by Stage of Development, Molecule Type and Program Type, 2018 60
Figure 13: Orphan and Rare Dermatology Drugs Market, Global, Pipeline for Key Indications by Stage of Development, 2018 61
Figure 14: Orphan and Rare Dermatology Drugs Market, Global, Pipeline for Key Indications by Molecule Type, 2018 62
Figure 15: Orphan and Rare Dermatology Drugs Market, Global, Overall Pipeline by Molecular Target and Stage of Development, 2018 64
Figure 16: Orphan and Rare Dermatology Drugs Market, Global, Pipeline for Key Indications by Molecular Target, 2018 65
Figure 17: Orphan and Rare Dermatology Drugs Market, Global, Clinical Trial Attrition Rates by Stage of Development (%), 2006-2018 69
Figure 18: Orphan and Rare Dermatology Drugs Market, Global, Clinical Trial Duration by Stage of Development (months), 2006-2018 69
Figure 19: Orphan and Rare Dermatology Drugs Market, Global, Clinical Trial Size by Stage of Development (participants), 2006-2018 70
Figure 20: Orphan and Rare Dermatology Drugs Market, Global, Clinical Program Size by Stage of Development (participants), 2006-2018 71
Figure 21: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for Epidiolex ($m), 2019-2024 73
Figure 22: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for Episalvan (birch bark extract) ($m), 2020-2024 74
Figure 23: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for KB-103 ($m), 2021-2024 75
Figure 24: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for Givosiran ($m), 2019-2024 76
Figure 25: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for EB-101 ($m), 2020-2024 78
Figure 26: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for OTL-103 ($m), 2021-2024 79
Figure 27: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for Siponimod ($m), 2021-2024 80
Figure 28: Orphan and Rare Dermatology Drugs Market, Global, Revenue Forecast for FCX-007 ($m), 2021-2024 81
Figure 29: Orphan and Rare Dermatology Drugs Market, Global, Premium Product Market Size ($bn), 2017-2024 83
Figure 30: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue Forecast for Products Acting on Interleukins ($m), 2017-2024 86
Figure 31: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue Forecast for Products Acting on TNF-α ($m), 2017-2024 87
Figure 32: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue Forecast for Products Acting on Tyrosine Protein Kinases ($m), 2019-2024 88
Figure 33: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue Forecast for Products Acting on Hormones and Receptors ($m), 2017-2024 89
Figure 34: Orphan and Rare Dermatology Drugs Market, Global, Company Analysis Matrix, 2017-2024 90
Figure 35: Orphan and Rare Dermatology Drugs Market, Global, Cluster by CAGR and Market Share, 2017-2024 91
Figure 36: Orphan and Rare Dermatology Drugs Market, Global, Forecast Market Share by Company (%), 2017-2024 95
Figure 37: Orphan and Rare Dermatology Drugs Market, Global, Companies by Compound Annual Growth Rate (%), 2017-2024 96
Figure 38: Orphan and Rare Dermatology Drugs Market, Global, Revenues by Route of Acquisition, 2017-2024 97
Figure 39: Orphan and Rare Dermatology Drugs Market, Global, Novartis Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2017-2024 98
Figure 40: Orphan and Rare Dermatology Drugs Market, Global, GW Pharmaceuticals Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2017-2024 99
Figure 41: Orphan and Rare Dermatology Drugs Market, Global, AbbVie Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2017-2024 100
Figure 42: Orphan Dermatology Drugs Market, Global, Roche Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2018-2024 101
Figure 43: Orphan and Rare Dermatology Drugs Market, Global, Pfizer Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2017-2024 102
Figure 44: Orphan and Rare Dermatology Drugs Market, Global, Amryt Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2017-2024 103
Figure 45: Orphan and Rare Dermatology Drugs Market, Global, GlaxoSmithKline Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2018-2024 104
Figure 46: Orphan and Rare Dermatology Drugs Market, Global, Celgene Orphan and Rare Dermatology Annual Revenue Forecast ($m), 2017-2024 105
Figure 47: Orphan and Rare Dermatology Drugs Market, Global, Late-Stage Pipeline Developers by Type, 2018 106
Figure 48: Orphan and Rare Dermatology Drugs Market, Global, Pipeline and Late-Stage Developers by Level of Orphan and Rare Dermatology Specialization, 2018 107
Figure 49: Orphan and Rare Dermatology Drugs Market, Global, Proportion of Total Company Revenue Attributed to Orphan and Rare Dermatology, 2024 108
Figure 50: Orphan and Rare Dermatology Drugs Market, Global, Licensing Deals by Region, Value and Year, 2006-2018 110
Figure 51: Orphan and Rare Dermatology Drugs Market, Global, Licensing Deals by Indication, 2006-2018 111
Figure 52: Orphan and Rare Dermatology Drugs Market, Global, Licensing Deals by Stage of Development and Value, 2006-2018 112
Figure 53: Orphan and Rare Dermatology Drugs Market, Global, Licensing Deals by Molecule Type and Molecular Target, 2006-2018 113
Figure 54: Orphan and Rare Dermatology Drugs Market, Global, Licensing Deals with Disclosed Values, 2006-2018 114
Figure 55: Orphan and Rare Dermatology Drugs Market, Global, Co-development Deals by Region, Year and Value, 2006-2018 115
Figure 56: Orphan and Rare Dermatology Drugs Market, Global, Co-development Deals by Key Indication, 2006-2018 116
Figure 57: Orphan and Rare Dermatology Drugs Market, Global, Co-development Deals by Stage of Development and Value, 2006-2018 117
Figure 58: Orphan and Rare Dermatology Drugs Market, Global, Co-development Deals by Molecule Type and Molecular Target, 2006-2018 118
Figure 59: Orphan and Rare Dermatology Drugs Market, Global, Co-development Deals with Disclosed Values, 2006-2018 119
Figure 60: Orphan and Rare Dermatology Drugs Market, Global, All Pipeline Products, 2018 (part 1) 129

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