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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

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

Summary


Dermatology is a highly diverse therapy area that deals with diseases of the skin, hair and nails. Over 3,000 distinct dermatological conditions are thought to exist, ranging from the rare diseases such as systemic sclerosis (scleroderma), an autoimmune disorder, to very common conditions such as acne vulgaris. The scope of dermatological disorders is diverse in terms of severity and clinical presentation. Many of these 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. As many dermatological conditions are chronic in nature, these symptoms can also increase the risk of developing psychosocial comorbidities - such as depression and anxiety - over time.

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.


1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Executive Summary
2.1 Neglected Therapy Area with High Levels of Genericization
2.2 Diverse Pipeline Demonstrates Potential to Address Unmet Needs
2.3 Increase in Strategic Consolidations in Recent Years
3 The Case for Innovation
3.1 Growing Opportunities for Biologic Products
3.2 Diversification of Molecular Targets
3.3 Innovative First-in-Class Product Developments Remain Attractive
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Innovation
3.5 Sustained Innovation in Orphan and Rare Dermatological Diseases
3.6 GBI Research Report Guidance
4 Clinical and Commercial Landscape
4.1 Overview of the Dermatology Market
4.2 Overview of Orphan and Rare Diseases
4.2.1 Overview of Systemic Sclerosis (Scleroderma)
4.2.2 Overview of Alopecia
4.2.3 Overview of Epidermolysis Bullosa
4.2.4 Overview of Pemphigus Vulgaris
4.2.5 Overview of Vitiligo
4.2.6 Overview of Cutaneous Lupus Erythematosus
4.3 Symptoms
4.3.1 Systemic sclerosis
4.3.2 Alopecia
4.3.3 Epidermolysis bullosa
4.3.4 Pemphigus Vulgaris
4.3.5 Vitiligo
4.3.6 Cutaneous Lupus Erythematosus
4.4 Diagnosis
4.4.1 Systemic sclerosis
4.4.2 Alopecia
4.4.3 Epidermolysis bullosa
4.4.4 Pemphigus Vulgaris
4.4.5 Vitiligo
4.4.6 Cutaneous Lupus Erythematosus
4.5 Etiology
4.5.1 Systemic Sclerosis
4.5.2 Alopecia
4.5.3 Epidermolysis Bullosa
4.5.4 Pemphigus Vulgaris
4.5.5 Vitiligo
4.5.6 Cutaneous Lupus Erythematosus
4.6 Pathophysiology
4.6.1 Systemic Sclerosis
4.6.2 Alopecia
4.6.3 Epidermolysis Bullosa
4.6.4 Pemphigus Vulgaris
4.6.5 Vitiligo
4.6.6 Cutaneous Lupus Erythematosus
4.7 Epidemiology
4.7.1 Systemic sclerosis
4.7.2 Alopecia
4.7.3 Epidermolysis Bullosa
4.7.4 Pemphigus Vulgaris
4.7.5 Vitiligo
4.7.6 Cutaneous Lupus Erythematosus
4.8 Co-morbidities and Complications
4.8.1 Systemic Sclerosis
4.8.2 Alopecia
4.8.3 Epidermolysis Bullosa
4.8.4 Pemphigus Vulgaris
4.8.5 Vitiligo
4.8.6 Cutaneous Lupus Erythematosus
4.9 Treatment
4.9.1 Systemic Sclerosis
4.9.2 Alopecia
4.9.3 Epidermolysis Bullosa
4.9.4 Pemphigus Vulgaris
4.9.5 Vitiligo
4.9.6 Cutaneous Lupus Erythematosus
4.10 Overview of Marketed Products
5 Assessment of Pipeline Product Innovation
5.1 Overview
5.2 Pipeline by Stage of Development and Molecule Type
5.2.1 Overall Pipeline
5.2.2 Pipeline by Key Indications
5.3 Pipeline by Molecular Target
5.3.1 Overall Pipeline
5.3.2 Pipeline for Key Indications
5.4 Comparative Distribution of Programs between the Market and Pipeline by Molecular Target Class
5.5 Comparative Distribution of First-in-Class and Non-First-in-Class Pipeline Programs by Molecular Target Class
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
5.7 Ratio of First-in-Class Programs to First-in-Class Molecular Targets by Stage of Development, Molecule Type and Molecular Target Class
5.8 List of All Pipeline Programs
6 Signaling Network, Disease Causation and Innovation Alignment
6.1 Complexity of Signaling Networks in Orphan and Rare Dermatological Diseases
6.2 Signaling Pathways and First-in-Class Molecular Target Integration
6.3 First-in-Class Molecular Target Matrix Assessment
7 First-in-Class Molecular Target Evaluation
7.1 Pipeline Programs Targeting Collagen type XVII (COL17A1)
7.2 Pipeline Programs Targeting Collagen type VII, alpha 1 (COL7A1)
7.3 Pipeline Programs Targeting Interleukin 2 Receptor Subunit Beta (IL2RB)
7.4 Pipeline Programs Targeting Desmoglein 1 (DSG1)
7.5 Pipeline Programs Targeting Nacht LRR and PYD Domains Containing Protein 3 (NLRP3)
7.6 Pipeline Programs Targeting Interleukin 3 Receptor Subunit Alpha (IL3RA)
7.7 Pipeline Programs Targeting Non-Receptor Tyrosine Protein Kinase TYK2 (TYK2)
7.8 Pipeline Programs Targeting Interleukin 6 Receptor Subunit Beta (IL6ST)
7.9 Pipeline Programs Targeting Potassium Voltage-Gated Channel Subfamily A Member 3 (KCNA3)
7.10 Pipeline Programs Targeting Kallikrein 7 (KLK7)
7.11 Pipeline Programs Targeting Eotaxin (CCL11)
7.12 Pipeline Programs Targeting Interleukin 2 (IL2)
8 Strategic Consolidations
8.1 Industry-wide First-in-Class Deals
8.2 Licensing Deals
8.2.1 Deals by Region, Value and Year
8.2.2 Deals by Key Indication and Value
8.2.3 Deals by Stage of Development and Value
8.2.4 Deals by Molecule Type and Molecular Target
8.2.5 List of Deals with Disclosed Deal Values
8.3 Co-development Deals
8.3.1 Deals by Region, Value and Year
8.3.2 Deals by Indication and Value
8.3.3 Deals by Stage of Development and Value
8.3.4 Deals by Molecule Type and Molecular Target
8.3.5 List of Deals with Disclosed Deal Values
8.4 First-in-Class Programs with and without Prior Licensing or Co-development Deal Involvement
9 Appendix
9.1 References
9.2 Abbreviations
9.3 Pipeline Disease List
9.4 Methodology
9.4.1 Data Integrity
9.4.2 Innovative and Meaningful Analytical Techniques and Frameworks
9.4.3 Evidence Based Analysis and Insight
9.5 Secondary Research
9.5.1 Market Analysis
9.5.2 Pipeline Analysis
9.5.3 Licensing and Co-development Deals
9.6 Contact Us
9.7 Disclaimer
1.1 List of Tables
Table 1: Orphan and Rare Dermatological Diseases, Global, Key Features of Collagen Type XVII (COL17A1), 2018
Table 2: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Collagen Type XVII (COL17A1), 2018
Table 3: Orphan and Rare Dermatological Diseases, Global, Key Features of Collagen Type VII, Alpha 1 (COL7A1), 2018
Table 4: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Collagen Type VII, Alpha 1 (COL7A1), 2018
Table 5: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 2 Receptor Subunit Beta (IL2RB), 2018
Table 6: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Interleukin 2 Receptor Subunit Beta (IL2RB), 2018
Table 7: Orphan and Rare Dermatological Diseases, Global, Key Features of Desmoglein 1 (DSG1), 2018
Table 8: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Desmoglein 1 (DSG1), 2018
Table 9: Orphan and Rare Dermatological Diseases, Global, Key Features of Nacht LRR and PYD Domains Containing Protein 3 (NLRP3), 2018
Table 10: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Nacht LRR and PYD Domains Containing Protein 3 (NLRP3), 2018
Table 11: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 3 Receptor Subunit Alpha (IL3RA), 2018
Table 12: Pipeline Programs Targeting Interleukin 3 Receptor Subunit Alpha (IL3RA), 2018
Table 13: Orphan and Rare Dermatological Diseases, Global, Key Features of Non-receptor Tyrosine Protein Kinase TYK2, 2018
Table 14: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Non-receptor Tyrosine Protein Kinase TYK2, 2018
Table 15: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 6 Receptor Subunit Beta (IL6ST), 2018
Table 16: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Interleukin 6 Receptor Subunit Beta (IL6ST), 2018
Table 17: Orphan and Rare Dermatological Diseases, Global, Key Features of Potassium Voltage Gated Subfamily A Member 3 (KCNA3), 2018
Table 18: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Potassium Voltage Gated Channel Subfamily A Member 3 (KCNA3), 2018
Table 19: Orphan and Rare Dermatological Diseases, Global, Key Features of Kallikrein 7 (KLK7), 2018
Table 20: Orphan and Rare Dermatological Disease, Global, Pipeline Programs Targeting Kallikrein 7 (KLK7), 2018
Table 21: Orphan and Rare Dermatological Diseases, Global, Key Features of Eotaxin (CCL11), 2018
Table 22: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Key Features of Eotaxin (CCL11), 2018
Table 23: Orphan and Rare Dermatological Diseases, Global, Key Features of Interleukin 2 (IL2), 2018
Table 24: Orphan and Rare Dermatological Diseases, Global, Pipeline Programs Targeting Interleukin 2 (IL2), 2018
1.2 List of Figures
Figure 1: Orphan and Rare Dermatological Diseases, US, Innovation Trends in Products Approvals, 1987-2014
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
Figure 3: Orphan and Rare Dermatological Diseases Global, Market by Molecule Type and Molecular Target Class, 2018
Figure 4: Orphan and Rare Dermatological Diseases, Global, Overall Pharmaceutical Pipeline by Therapy Area, 2018
Figure 5: Orphan and Rare Dermatological Diseases, Global, Overall Pipeline by Stage of Development and Molecule Type, 2018
Figure 6: Orphan and Rare Dermatological Diseases, Global, Pipeline for Key Indications by Stage of Development, 2018
Figure 7: Orphan and Rare Dermatological Diseases, Global, Pipeline for Key Indications by Molecule Type, 2018
Figure 8: Orphan and Rare Dermatological Diseases, Global, Overall Pipeline by Molecular Target and Stage of Development, 2018
Figure 9: Orphan and Rare Dermatological Diseases, Global, Pipeline for Key Indications by Molecular Target Class, 2018
Figure 10: Orphan and Rare Dermatological Diseases, Global, Pipeline and Marketed Programs by Molecular Target Class, 2018
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
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
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
Figure 14: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 1)
Figure 15: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 2)
Figure 16: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 3)
Figure 17: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 4)
Figure 18: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 5)
Figure 19: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 6)
Figure 20: Orphan and Rare Dermatological Diseases, Global, All First-in-Class Pipeline Programs, 2018 (part 7)
Figure 21: Orphan and Rare Dermatological Diseases, Global, First-in-Class Molecular Target Matrix Assessment, 2018 (part 1)
Figure 22: Orphan and Rare Dermatological Diseases, Global, First-in-Class Molecular Target Matrix Assessment, 2018 (part 2)
Figure 23: Orphan and Rare Dermatological Diseases, Global, First-in-Class Molecular Target Matrix Assessment, 2018 (part 3)
Figure 24: Orphan Dermatological Disease, Global, Industry-Wide Licensing Deals by First-in-Class Status and Stage of Development, 2006-2015
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
Figure 26: Orphan and Rare Dermatological Diseases, Global, Licensing Deals by Region, Value and Year, 2006-2018
Figure 27: Orphan and Rare Dermatological Diseases, Global, Number and Aggregate Deal Value of Licensing Deals for Systemic Sclerosis, Alopecia and Epidermolysis Bullosa, 2018
Figure 28: Orphan and Rare Dermatological Diseases, Global, Licensing Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006-2018
Figure 29: Orphan and Rare Dermatological Diseases, Global, Number and Aggregate Deal Value of Licensing Deals by Molecule Type, 2006-2018
Figure 30: Orphan and Rare Dermatological Diseases Global, Licensing Deals with Disclosed Deal Values, 2006-2018
Figure 31: Orphan and Rare Dermatological Diseases, Global, Co-development Deals by Region, Value and Year, 2006-2018
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
Figure 33: Orphan Dermatological Diseases, Co-development Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006-2018
Figure 34: Orphan Dermatological Diseases, Global, Number and Aggregate Deal Value of Co-development Deals by Molecule Type and Molecular Target Class, 2006-2018
Figure 35: Orphan and Rare Dermatological Disease, Global, Co-development Deals with Disclosed Deal Values, 2006-2018
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)
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)

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