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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

  • Published: Sep-2018
  • Report Code: GBIHC483MR
  • Report Format: pdf


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.


  • 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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