Porphyria is a rare disorder that is characterized by excessive accumulation of porphyrin, a compound that aids in the formation of heme (an essential part of hemoglobin that helps carry oxygen in blood). Any anomaly caused by genetic or acquired abnormalities in heme biosynthesis (produced majorly in bone marrow and liver) can result in toxicity. It is worth highlighting that, till date, more than 1,000 mutations that can cause porphyria have been identified. However, prevalence of porphyria still remains unknown. Several treatment options such as gene therapy, proteasome inhibition and pharmacologic chaperones are currently being investigated among various other targeted treatment options.
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Key Market Insights
15+ therapies have been / are being developed for the treatment of different types of porphyria
More than 70% of the aforementioned candidates are currently under clinical evaluation. Further, three therapies, namely Panhematin™, GIVLAARI® and SCENESSE®, have already been approved for the treatment of different types of porphyria.
More than 45% of the players evaluating therapies for porphyria are small companies
North America has emerged as a key hub for the development of porphyria therapies, featuring the presence of 65% developers. The developer landscape is further dominated by players that have been established between 2001-2010, representing around 45% of the total number of stakeholders.
A number of clinical trials evaluating therapies for porphyria, have been registered
Majority of the clinical studies have been completed. More than 30% of the overall trials are phase I studies. Further, it is worth noting that, most of the trials (~ 60%) focused on porphyria therapies were registered post-2010.
Partnership activity in this field has increased at a CAGR of 9.6%, between 2018 and 2020
More than 70% of the reported deals were established post-2018, with the maximum activity being reported in 2019 and 2020. Majority of the instances captured in the report were product distribution / commercialization agreements (~45%).
380+ articles have been published related to porphyria, since January 2018
Close to 20% publications mentioned in the report were focused on the assessment of therapeutics that have been / are being developed for the treatment of erythropoietic protoporphyria. Example of prominent journals include (in decreasing order of number of publications) Molecular Genetics, Orphanet Journal of Rare Diseases, British Journal of Dermatology and Molecular Genetics, and Metabolism Reports.
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Key Questions Answered
- What are the prevalent R&D trends related to Porphyria?
- What are the key challenges faced by stakeholders engaged in this domain?
- What are the principal therapies developed by the companies in this domain?
- Who are the leading industry and non-industry players in this market?
- What are the key geographies where research on porphyria is actively being conducted?
- Who are the key investors in this domain?
- Who are the key opinion leaders / experts in this field?
- What kind of partnership models are commonly adopted by industry stakeholders?
- What are the factors that are likely to influence the evolution of this upcoming market?
- How is the current and future market opportunity likely to be distributed across key market segments?
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