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