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
Roots Analysis
has announced the addition of “Porphyria
Targeting Therapies Market, 2021-2030” report to its list of offerings.
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.
To
order this 130+ slides report, which features 90+ figures, please visit https://www.rootsanalysis.com/reports/porphyria-pipline-review.html
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.
Over
30% of the therapies have been / are being developed for erythropoietic
protoporphyria
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.
To request a sample copy / brochure of this report,
please visit this link.
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:
§ Drug
§ GIVLAARI®
§ Panhematin®
§ SCENESSE®
§ MT-7117
§ Colestid
§ HARVONI®
§ Type
of Porphyria
- Acute
Hepatic Porphyria
- Acute
Intermittent Porphyria
- Erythropoietic
Protoporphyria
- Hereditary
Coproporphyria
- Porphyria
Cutanea Tarda
- Variegate
Porphyria
- X-Linked
Porphyria
§ Route
of Administration
- Oral
- Intravenous
- Subcutaneous
§ Key
Geographical Regions
- North
America
- Europe
- Asia-Pacific
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
For additional details,
please visit https://www.rootsanalysis.com/reports/porphyria-pipline-review.html or email sales@rootsanalysis.com
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Contact:
Ben Johnson
+1 (415) 800 3415
+44 (122) 391 1091
Ben.johnson@rootsanalysis.com
