Several novel and innovative therapeutic approaches, both small molecule and biologics, are being currently investigated at various phases of development in order to avoid the beginning of disease-induced attacks and other long-term effects of porphyria
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.
Around 50% of the therapeutics are being developed as biologics
Majority (over 65%) of the abovementioned biologic drugs have been / are being designed for administration via the intravenous route. Furthermore, majority of the drugs (37%) have been / are being targeting acute intermittent porphyria.
More than 65% the abovementioned therapies are currently being evaluated in clinical phases. Further, around 60% of the aforementioned therapy candidates are being developed as small molecules.
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.
Around 15 eminent individuals were identified as key opinion leaders (KOLs) in this domain
More than 65% of these KOLs were observed to be associated with organizations based in US, followed by those affiliated to institutes in Spain (20%) and South Africa (7%). Further, over 65% of the KOLs are currently affiliated to academic institutes, such as schools and universities.
North America is anticipated to capture over 60% of the global market share in 2030
In 2030, more than 50% of the market revenues are expected to be generated from sales of therapeutics intended for the treatment of erythropoietic protoporphyria and porphyria cutanea tarda. Further, therapies designed for oral route of administration are expected to occupy a larger share (51%) of the overall market, in the foreseen future.
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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?
The financial opportunity within the porphyria therapies market has been analyzed across the following segments:
- Type of Porphyria
- Acute Hepatic Porphyria
- Acute Intermittent Porphyria
- Erythropoietic Protoporphyria
- Hereditary Coproporphyria
- Porphyria Cutanea Tarda
- Variegate Porphyria
- X-Linked Porphyria
- Route of Administration
- Key Geographical Regions
- North America
The research includes profiles of key players (listed below); each profile features a brief overview of company, pipeline details, recent developments (including collaborations and expansions) and an informed future outlook.
- Agios Pharmaceutical
- Alnylam Pharmaceuticals
- Clinuvel Pharmaceuticals
- Disc Medicine
- Mitsubishi Tanabe Pharma
- Moderna Therapeutics
- Palatin Technologies
- Recordati Rare Diseases
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