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 ($m), 2017-2024 84 Table 16: Orphan and Rare Dermatology Drugs Market, Global, Forecast Revenue by Company (%), 2017-2024 931.2 List of Figures Figure 1: Orphan and Rare Dermatology Drugs Market, 7MM, Epidemiology Patterns for Systemic Sclerosis (Scleroderma), 2017-2024 26 Figure 2: Orphan and Rare Dermatology Drugs Market, Global, Annual Revenue for Humira ($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