Frontier Pharma: Orphan and Rare Dermatological Diseases - First-in-Class Therapies Demonstrate Potential Disease-Modifying Effects in Areas of High Unmet Need Such as Epidermolysis Bullosa

GBI Research
86 Pages - GBI10604
$6,995.00

Summary

Orphan and rare dermatological diseases are considerably diverse in terms of pathophysiology, clinical presentation and disease severity. Many of these conditions are extremely debilitating and can even be life-threatening in some cases.

Disease visibility can also have a profoundly negative impact on patient confidence. Unmet need within the orphan and rare dermatology market is extremely high with some diseases having no effective treatments. The market is highly genericized and dominated to a large extent by products acting on hormones and their receptors.

Most of these are corticosteroids and are used for symptom management with no disease-modifying effects. There is a significant unmet need for more efficacious disease-modifying treatments and safer treatment options across orphan and rare dermatological diseases, as physicians often cite poor efficacy, low patient compliance and problematic safety profiles as issues associated with the long-term use of available treatments.

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

Scope

- There is a strong need for innovative new therapies across the orphan and rare dermatology market. How are orphan and rare dermatological diseases currently managed? What are the greatest unmet needs within this market?
- There are 262 pipeline products in development across all orphan and rare dermatological diseases. How does the composition of the pipeline compare with that of the existing market? Which molecular targets are most frequently acted upon by pipeline drugs? How do products in development for the key indications differ in terms of molecule type?
- Over one-third of pipeline products with a disclosed molecular target are first-in-class. How does the proportion of first-in-class products in development differ in terms of stage of development, molecule type and molecular target class? Which are the most promising first-in-class targets?
- The deals landscape has become more active in recent years. Which indications and products have attracted the highest deal values?

Reasons to buy

This report will allow you to:
- Appreciate the current clinical and commercial landscapes by considering disease pathogenesis, etiology, epidemiology, symptoms, diagnosis and treatment options.
- Visualize the composition of the market in terms of dominant molecule types and molecular targets, highlighting what the current unmet needs are and how they can be addressed. This knowledge allows a competitive understanding of gaps in the current market.
- Recognize innovative pipeline trends by analyzing therapies by stage of development, molecule type and molecular target.
- Assess the therapeutic potential of first-in-class molecular targets. Using a proprietary matrix, first-in-class targets have been assessed and ranked according to clinical potential. Promising first-in-class targets have been reviewed in greater detail.
- Consider first-in-class pipeline products with no prior involvement in licensing and co-development deals that may represent potential investment opportunities.

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1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 4
1.2 List of Figures 4
2 Executive Summary 6
2.1 Neglected Therapy Area with High Levels of Genericization 6
2.2 Diverse Pipeline Demonstrates Potential to Address Unmet Needs 6
2.3 Increase in Strategic Consolidations in Recent Years 6
3 The Case for Innovation 7
3.1 Growing Opportunities for Biologic Products 8
3.2 Diversification of Molecular Targets 8
3.3 Innovative First-in-Class Product Developments Remain Attractive 8
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Innovation 9
3.5 Sustained Innovation in Orphan and Rare Dermatological Diseases 9
3.6 GBI Research Report Guidance 9
4 Clinical and Commercial Landscape 11
4.1 Overview of the Dermatology Market 11
4.2 Overview of Orphan and Rare Diseases 11
4.2.1 Overview of Systemic Sclerosis (Scleroderma) 12
4.2.2 Overview of Alopecia 12
4.2.3 Overview of Epidermolysis Bullosa 12
4.2.4 Overview of Pemphigus Vulgaris 13
4.2.5 Overview of Vitiligo 13
4.2.6 Overview of Cutaneous Lupus Erythematosus 13
4.3 Symptoms 13
4.3.1 Systemic sclerosis 13
4.3.2 Alopecia 14
4.3.3 Epidermolysis bullosa 14
4.3.4 Pemphigus Vulgaris 14
4.3.5 Vitiligo 14
4.3.6 Cutaneous Lupus Erythematosus 14
4.4 Diagnosis 14
4.4.1 Systemic sclerosis 15
4.4.2 Alopecia 15
4.4.3 Epidermolysis bullosa 16
4.4.4 Pemphigus Vulgaris 16
4.4.5 Vitiligo 16
4.4.6 Cutaneous Lupus Erythematosus 16
4.5 Etiology 16
4.5.1 Systemic Sclerosis 16
4.5.2 Alopecia 16
4.5.3 Epidermolysis Bullosa 17
4.5.4 Pemphigus Vulgaris 17
4.5.5 Vitiligo 17
4.5.6 Cutaneous Lupus Erythematosus 18
4.6 Pathophysiology 18
4.6.1 Systemic Sclerosis 19
4.6.2 Alopecia 19
4.6.3 Epidermolysis Bullosa 19
4.6.4 Pemphigus Vulgaris 19
4.6.5 Vitiligo 19
4.6.6 Cutaneous Lupus Erythematosus 20
4.7 Epidemiology 20
4.7.1 Systemic sclerosis 20
4.7.2 Alopecia 20
4.7.3 Epidermolysis Bullosa 21
4.7.4 Pemphigus Vulgaris 21
4.7.5 Vitiligo 21
4.7.6 Cutaneous Lupus Erythematosus 21
4.8 Co-morbidities and Complications 21
4.8.1 Systemic Sclerosis 22
4.8.2 Alopecia 22
4.8.3 Epidermolysis Bullosa 22
4.8.4 Pemphigus Vulgaris 22
4.8.5 Vitiligo 22
4.8.6 Cutaneous Lupus Erythematosus 22
4.9 Treatment 22
4.9.1 Systemic Sclerosis 23
4.9.2 Alopecia 24
4.9.3 Epidermolysis Bullosa 24
4.9.4 Pemphigus Vulgaris 24
4.9.5 Vitiligo 25
4.9.6 Cutaneous Lupus Erythematosus 25
4.10 Overview of Marketed Products 25
5 Assessment of Pipeline Product Innovation 27
5.1 Overview 27
5.2 Pipeline by Stage of Development and Molecule Type 27
5.2.1 Overall Pipeline 27
5.2.2 Pipeline by Key Indications 28
5.3 Pipeline by Molecular Target 32
5.3.1 Overall Pipeline 32
5.3.2 Pipeline for Key Indications 33
5.4 Comparative Distribution of Programs between the Market and Pipeline by Molecular Target Class 35
5.5 Comparative Distribution of First-in-Class and Non-First-in-Class Pipeline Programs by Molecular Target Class 35
5.6 Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Programs by Stage of Development, Molecule Type and Molecular Target Class 37
5.7 Ratio of First-in-Class Programs to First-in-Class Molecular Targets by Stage of Development, Molecule Type and Molecular Target Class 38
5.8 List of All Pipeline Programs 40
6 Signaling Network, Disease Causation and Innovation Alignment 47
6.1 Complexity of Signaling Networks in Orphan and Rare Dermatological Diseases 47
6.2 Signaling Pathways and First-in-Class Molecular Target Integration 47
6.3 First-in-Class Molecular Target Matrix Assessment 47
7 First-in-Class Molecular Target Evaluation 52
7.1 Pipeline Programs Targeting Collagen type XVII (COL17A1) 52
7.2 Pipeline Programs Targeting Collagen type VII, alpha 1 (COL7A1) 52
7.3 Pipeline Programs Targeting Interleukin 2 Receptor Subunit Beta (IL2RB) 54
7.4 Pipeline Programs Targeting Desmoglein 1 (DSG1) 55
7.5 Pipeline Programs Targeting Nacht LRR and PYD Domains Containing Protein 3 (NLRP3) 56
7.6 Pipeline Programs Targeting Interleukin 3 Receptor Subunit Alpha (IL3RA) 58
7.7 Pipeline Programs Targeting Non-Receptor Tyrosine Protein Kinase TYK2 (TYK2) 59
7.8 Pipeline Programs Targeting Interleukin 6 Receptor Subunit Beta (IL6ST) 61
7.9 Pipeline Programs Targeting Potassium Voltage-Gated Channel Subfamily A Member 3 (KCNA3) 62
7.10 Pipeline Programs Targeting Kallikrein 7 (KLK7) 63
7.11 Pipeline Programs Targeting Eotaxin (CCL11) 64
7.12 Pipeline Programs Targeting Interleukin 2 (IL2) 65
8 Strategic Consolidations 67
8.1 Industry-wide First-in-Class Deals 67
8.2 Licensing Deals 68
8.2.1 Deals by Region, Value and Year 68
8.2.2 Deals by Key Indication and Value 69
8.2.3 Deals by Stage of Development and Value 70
8.2.4 Deals by Molecule Type and Molecular Target 71
8.2.5 List of Deals with Disclosed Deal Values 72
8.3 Co-development Deals 73
8.3.1 Deals by Region, Value and Year 73
8.3.2 Deals by Indication and Value 74
8.3.3 Deals by Stage of Development and Value 74
8.3.4 Deals by Molecule Type and Molecular Target 74
8.3.5 List of Deals with Disclosed Deal Values 76
8.4 First-in-Class Programs with and without Prior Licensing or Co-development Deal Involvement 76
9 Appendix 79
9.1 References 79
9.2 Abbreviations 83
9.3 Pipeline Disease List 84
9.4 Methodology 84
9.4.1 Data Integrity 84
9.4.2 Innovative and Meaningful Analytical Techniques and Frameworks 85
9.4.3 Evidence Based Analysis and Insight 85
9.5 Secondary Research 85
9.5.1 Market Analysis 85
9.5.2 Pipeline Analysis 85
9.5.3 Licensing and Co-development Deals 86
9.6 Contact Us 86
9.7 Disclaimer 86

1.1 List of Tables
Table 1: Orphan and Rare Dermatological Diseases, Global, Key Features of Collagen Type XVII (COL17A1), 2018 52
Table 2: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Collagen Type XVII (COL17A1), 2018 52
Table 3: Orphan and Rare Dermatological Diseases, Global, Key Features of Collagen Type VII, Alpha 1 (COL7A1), 2018 53
Table 4: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Collagen Type VII, Alpha 1 (COL7A1), 2018 54
Table 5: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 2 Receptor Subunit Beta (IL2RB), 2018 55
Table 6: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Interleukin 2 Receptor Subunit Beta (IL2RB), 2018 55
Table 7: Orphan and Rare Dermatological Diseases, Global, Key Features of Desmoglein 1 (DSG1), 2018 56
Table 8: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Desmoglein 1 (DSG1), 2018 56
Table 9: Orphan and Rare Dermatological Diseases, Global, Key Features of Nacht LRR and PYD Domains Containing Protein 3 (NLRP3), 2018 57
Table 10: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Nacht LRR and PYD Domains Containing Protein 3 (NLRP3), 2018 58
Table 11: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 3 Receptor Subunit Alpha (IL3RA), 2018 59
Table 12: Pipeline Programs Targeting Interleukin 3 Receptor Subunit Alpha (IL3RA), 2018 59
Table 13: Orphan and Rare Dermatological Diseases, Global, Key Features of Non-receptor Tyrosine Protein Kinase TYK2, 2018 60
Table 14: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Non-receptor Tyrosine Protein Kinase TYK2, 2018 61
Table 15: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 6 Receptor Subunit Beta (IL6ST), 2018 62
Table 16: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Interleukin 6 Receptor Subunit Beta (IL6ST), 2018 62
Table 17: Orphan and Rare Dermatological Diseases, Global, Key Features of Potassium Voltage Gated Subfamily A Member 3 (KCNA3), 2018 63
Table 18: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Potassium Voltage Gated Channel Subfamily A Member 3 (KCNA3), 2018 63
Table 19: Orphan and Rare Dermatological Diseases, Global, Key Features of Kallikrein 7 (KLK7), 2018 64
Table 20: Orphan and Rare Dermatological Disease, Global, Pipeline Programs Targeting Kallikrein 7 (KLK7), 2018 64
Table 21: Orphan and Rare Dermatological Diseases, Global, Key Features of Eotaxin (CCL11), 2018 65
Table 22: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Key Features of Eotaxin (CCL11), 2018 65
Table 23: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 2 (IL2), 2018 66
Table 24: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Interleukin 2 (IL2), 2018 66

1.2 List of Figures
Figure 1: Orphan and Rare Dermatological Diseases, US, Innovation Trends in Products Approvals, 1987–2014 7
Figure 2: Orphan and Rare Dermatological Diseases, US, Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006–2013 9
Figure 3: Orphan and Rare Dermatological Diseases Global, Market by Molecule Type and Molecular Target Class, 2018 26
Figure 4: Orphan and Rare Dermatological Diseases, Global, Overall Pharmaceutical Pipeline by Therapy Area, 2018 27
Figure 5: Orphan and Rare Dermatological Diseases, Global, Overall Pipeline by Stage of Development and Molecule Type, 2018 28
Figure 6: Orphan and Rare Dermatological Diseases, Global, Pipeline for Key Indications by Stage of Development, 2018 29
Figure 7: Orphan and Rare Dermatological Diseases, Global, Pipeline for Key Indications by Molecule Type, 2018 31
Figure 8: Orphan and Rare Dermatological Diseases, Global, Overall Pipeline by Molecular Target and Stage of Development, 2018 33
Figure 9: Orphan and Rare Dermatological Diseases, Global, Pipeline for Key Indications by Molecular Target Class, 2018 34
Figure 10: Orphan and Rare Dermatological Diseases, Global, Pipeline and Marketed Programs by Molecular Target Class, 2018 35
Figure 11: Orphan and Rare Dermatological Diseases, Global, First-in-Class and Non-First-in-Class Pipeline Programs by Molecular Target Class and Stage of Development, 2018 36
Figure 12: Orphan and Rare Dermatological Diseases, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Programs by Stage of Development and Molecular Target Class, 2018 38
Figure 13: Orphan and Rare Dermatological Diseases, Global, Ratio of First-in-Class Programs to First-in-Class Targets by Stage of Development and Molecular Target Class, 2018 39
Figure 14: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 1) 40
Figure 15: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 2) 41
Figure 16: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 3) 42
Figure 17: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 4) 43
Figure 18: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 5) 44
Figure 19: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 6) 45
Figure 20: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 7) 46
Figure 21: Orphan and Rare Dermatological Diseases, Global, First-in-Class Molecular Target Matrix Assessment, 2018 (part 1) 49
Figure 22: Orphan and Rare Dermatological Diseases, Global, First-in-Class Molecular Target Matrix Assessment, 2018 (part 2) 50
Figure 23: Orphan and Rare Dermatological Diseases, Global, First-in-Class Molecular Target Matrix Assessment, 2018 (part 3) 51
Figure 24: Orphan Dermatological Disease, Global, Industry-Wide Licensing Deals by First-in-Class Status and Stage of Development, 2006–2015 67
Figure 25: Orphan Dermatological Diseases, Global, Industry-Wide Licensing Deals by First-in-Class Status, Deal Value, Upfront Payment Value and Stage of Development, 2006–2014 68
Figure 26: Orphan and Rare Dermatological Diseases, Global, Licensing Deals by Region, Value and Year, 2006–2018 69
Figure 27: Orphan and Rare Dermatological Diseases, Global, Number and Aggregate Deal Value of Licensing Deals for Systemic Sclerosis, Alopecia and Epidermolysis Bullosa, 2018 70
Figure 28: Orphan and Rare Dermatological Diseases, Global, Licensing Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006–2018 70
Figure 29: Orphan and Rare Dermatological Diseases, Global, Number and Aggregate Deal Value of Licensing Deals by Molecule Type, 2006–2018 71
Figure 30: Orphan and Rare Dermatological Diseases Global, Licensing Deals with Disclosed Deal Values, 2006–2018 72
Figure 31: Orphan and Rare Dermatological Diseases, Global, Co-development Deals by Region, Value and Year, 2006–2018 73
Figure 32: Orphan and Rare Dermatological Diseases, Global, Number and Aggregate Deal Value of Co-development Deals for Systemic Sclerosis, Alopecia and Epidermolysis Bullosa, 2018 74
Figure 33: Orphan Dermatological Diseases, Co-development Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006–2018 74
Figure 34: Orphan Dermatological Diseases, Global, Number and Aggregate Deal Value of Co-development Deals by Molecule Type and Molecular Target Class, 2006–2018 75
Figure 35: Orphan and Rare Dermatological Disease, Global, Co-development Deals with Disclosed Deal Values, 2006–2018 76
Figure 36: Orphan and Rare Dermatological Diseases, Global, First-in-Class Programs with and without Prior Involvement in Licensing or Co-development Deals, 2018 (part 1) 77
Figure 37: Orphan and Rare Dermatological Diseases, Global, First-in-Class Programs with and without Prior Involvement in Licensing or Co-development Deals, 2018 (part 2) 78

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