Overview
The “Porphyria: Pipeline Review, Developer Landscape and Competitive Insights, 2021-2030” report features an extensive study on the marketed, clinical and preclinical molecules available/being developed for the treatment of Porphyria.
This report provides an in-depth analysis of the product pipeline and developer companies, highlighting the current treatment practices, emerging drugs, and market share of the individual therapies. In addition to other elements, the study includes detailed assessment of the current market landscape, providing information about the product’s mechanism of action, dosage and administration, and pharmacological studies, regulatory milestones, and other development activities.
One of the key objectives of the report was to estimate the existing market size and the future opportunity of therapies for Porphyria, over the next decade. Based on multiple parameters, such as disease prevalence, anticipated adoption of the forecasted therapies and the likely selling price of such therapeutic products, we have provided informed estimates on the evolution of the market for the period 2021-2030.
Scope of the Report
The “Porphyria Targeting Therapies Market by Type of Drug Class (Biologics and Small Molecules), Type of Porphyria (Acute Hepatic Porphyria, Acute Intermittent Porphyria, Erythropoietic Protoporphyria, Hereditary Porphyria, Porphyria Cutanea Tarda, Variegate Porphyria and X-Linked Protoporphyria), Route of Administration (Oral, Intravenous and Subcutaneous) and Key Geographical Regions (US, Canada, UK, France, Germany, Italy, Spain, Netherlands, Australia and Japan): Industry Trends and Global Forecasts: Industry Trends and Global Forecasts, 2021-2030” report features an extensive study of the current market landscape and future opportunity for the players engaged in developing porphyria targeting therapies. The study presents an in-depth analysis, highlighting the capabilities of various developers engaged in this domain.
Amongst other elements, the report features:
- A detailed review of the current market landscape of porphyria targeting therapies providing information on phase of development, drug class (biologic, small molecule), route of administration (oral, intravenous, subcutaneous), mechanism of action, type of porphyria (Acute Hepatic Porphyria, Acute Intermittent Porphyria, Congenital Erythropoietic Porphyria, Erythropoietic Protoporphyria, Hereditary Coproporphyria, Porphyria Cutanea Tarda, Variegate Porphyria, X-Linked Protoporphyria), drug designations (breakthrough therapy designation, fast track, orphan drug designation)
- A detailed review of the current market landscape of porphyria targeting therapies developers providing information on year of establishment, headquarters, company size (in terms of employee count), type of player (industry, non-industry) and ownership (public, private, non-profit)
- An insightful case study on terminated and dormant programs highlighting the terminated and dormant clinical studies in this domain.
- An in-depth clinical trial analysis providing information on trial status, trial registration year, trial phase, number of patients enrolled, geographical distribution, type of treatment, type of sponsors, active players, study design, type of allocation, type of intervention and type of porphyria.
- An insightful key opinion leader analysis highlighting the key principal investigators, appointed to lead clinical investigations and examine therapies targeting porphyria using our proprietary scoring based on parameters such as number of publication, number of citations, number of clinical trials, work experience, number of affiliated companies, number of LinkedIn connections and number of affiliated organizations.
- An insightful competitiveness analysis of the players engaged in the development of therapies targeting porphyria, taking into consideration their portfolio strength (in terms number of drugs in pipeline, phase of development and drug class), number of partnerships, patent portfolio, google hits, year of establishment and size of the company.
- An analysis of publications that have been published related to this domain based on key parameters such as year of publication, top authors, top journal and emerging focus area.
- An analysis of the partnerships that have been inked by stakeholders in this domain, in the period between 2010 and 2020, covering product development and commercialization agreement, clinical trial agreement, product development agreement, product licensing agreement and other relevant types of deals.
- Elaborate profiles of key players developing porphyria targeting therapies. Each company profile features a brief overview of the company (including information on year of establishment, number of employees, location of headquarters and key members of the executive team, financial/funding information and product portfolio), recent developments and an informed future outlook.
One of the key objectives of this report was to estimate the existing market size and to estimate the opportunity associated with the porphyria market We have developed informed estimates on the financial evolution of the market, over the period 2021-2030. The report also provides details on the likely distribution of the current and forecasted opportunity across [A] drug class (biologics and small molecules), [B] route of administration (oral, intravenous and subcutaneous), [C] type of porphyria (Acute Hepatic Porphyria, Acute Intermittent Porphyria, Erythropoietic Porphyria, Hereditary Porphyria, Porphyria Cutanea Tarda, Variegate Porphyria And X-Linked Porphyria) , [D] regional distribution (US, Canada, UK, Germany, France, Italy, Spain, Netherlands, Australia and Japan),. In order to account for future uncertainties and to add robustness to our forecast model, we have provided three scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.
All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified.
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 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?
Table of Contents
1. BACKGROUND
1.1. Context
1.2. Project Report
2. EXECUTIVE SUMMARY
3. INTRODUCTION
4. MARKET LANDSCAPE
4.1. Market Landscape of Porphyria Drugs
4.2. Market Landscape of Porphyria Drug Developers
5. CASE STUDY
5.1 Case Study on Terminated and Dormant Programs
6. KEY MARKET INSIGHTS
6.1. Clinical Trial Analysis
6.2. KOL Analysis
6.3. Company Competitiveness Analysis
6.4. Publication Analysis
6.6 Partnership and Collaborations
7. COMPANY PROFILES
7.1. Agios Pharmaceuticals
7.2. Alnylam Pharmaceuticals
7.3. Clinuvel Pharmaceuticals
7.4. Mitsubishi Tanabe Pharma
7.5. Moderna Therapeutics
7.6. Palatin Technologies
7.7. Recordati Rare Diseases
8. MARKET FORECAST
8.1. Drugs Forecasted and Sales Estimated
8.1.1. GIVLAARI®
8.1.2. Panhematin™
8.1.3. SCENESSE®
8.1.4. MT-7117
8.1.5. Colestid
8.1.6. HARVONI®
8.2. Global Market Forecast, Till 2030
8.3. Distribution by Drug Class
8.4. Distribution by Type of Porphyria
8.5. Distribution by Route of Administration
8.6. Distribution by Region
9. APPENDICES
9.1. Clinical Trial Analysis
9.2. Publication Analysis
9.3. List of Figures
9.4. List of Companies and Organizations
9.2. Publication Analysis
9.3. List of Figures
9.4. List of Companies and Organizations
List Of Figures
Figure 1.1 Distribution by Phase of Development
Figure 1.2 Distribution by Drug Class
Figure 1.3 Distribution by Phase of Development and Drug Class
Figure 1.4 Distribution by Type of Porphyria
Figure 1.5 Distribution by Type of Porphyria and Phase of Development
Figure 1.6 Distribution by Route of Administration
Figure 1.7 Distribution by Mechanism of Action
Figure 1.8 Distribution by Drug Designation Obtained
Figure 1.9 Distribution by Type of Player and Ownership
Figure 1.10 Distribution by Company Size
Figure 1.11 Distribution by Year of Establishment
Figure 1.12 Players in North America
Figure 1.13 Players in Europe
Figure 1.14 Players in Rest of the World
Figure 1.15 Distribution by Phase of Development and Location of Headquarters
Figure 1.16 Distribution by Type of Molecule and Location of Headquarters
Figure 1.17 Distribution by Phase of Development and Type of Molecule
Figure 3.1 Distribution by Trial Registration Year
Figure 3.2 Distribution by Trial Phase
Figure 3.3 Distribution by Trial Status
Figure 3.4 Analysis by Type of Sponsor
Figure 3.5 Key Industry Players
Figure 3.6 Key Non-industry Players
Figure 3.7 Distribution by Type of Porphyria
Figure 3.8 Distribution by Type of Sponsor and Type of Porphyria
Figure 3.9 Distribution by Trial Status and Type of Porphyria
Figure 3.10 Distribution by Enrolled Patients, Trial Status and Geography
Figure 3.11 Distribution by Trial Status and Geography
Figure 3.12 Distribution of Number of Trials by Geography
Figure 4.1 Distribution by Type of Organization
Figure 4.2 Distribution by Region
Figure 4.3 Distribution by Qualification
Figure 4.4 Distribution by Principal Investigator and Type of Porphyria
Figure 4.5 Distribution by Type of Porphyria
Figure 4.6 KOL Activeness V/S Strength: Scatter Plot
Figure 4.7 Publisher vs Third Party Score
Figure 4.8 Publisher vs Third Party Score 2
Figure 4.9 Regional Distribution
Figure 4.10 Activeness of KOLs Investigating Drugs in Clinical Studies
Figure 5.1 Benchmarking of Prominent Companies
Figure 5.2 Distribution by Developers
Figure 6.1 Distribution by Year of Publication
Figure 6.2 Distribution by Type of Porphyria
Figure 6.3 Distribution by Type of Porphyria and Year of Publication
Figure 6.4 Key Journals based on Publications Count
Figure 6.5 Word Cloud: Emerging Focus Areas
Figure 6.6 Distribution by Most Popular Authors
Figure 7.1 Distribution by Year of Collaboration
Figure 7.2 Distribution by Type of Collaboration
Figure 7.3 Distribution by Year of Collaboration and Type of Collaboration
Figure 7.4 Distribution by Key Players
Figure 7.5 Key Drugs by Number of Partnerships
Figure 7.6 Intercontinental and Intracontinental Agreements
Figure 9.1 GIVLAARI®: Global Sales Forecast, 2021-2030
Figure 9.2 GIVLAARI®: Regional Sales Forecast, 2021-2030
Figure 9.3 Panhematin®: Global Sales Forecast, 2021-2030
Figure 9.4 Panhematin®: Regional Sales Forecast, 2021-2030
Figure 9.5 SCENESSE®: Global Sales Forecast, 2021-2030
Figure 9.6 SCENESSE®: Regional Sales Forecast, 2021-2030
Figure 9.7 MT-7117: Global Sales Forecast, 2022-20301
Figure 9.8 MT-7117: Regional Sales Forecast, 2022-20301
Figure 9.9 Colestid: Global Sales Forecast, 2022-2030
Figure 9.10 Colestid: Regional Sales Forecast, 2022-2030
Figure 9.11 HARVONI®: Global Sales Forecast, 2025-2030
Figure 9.12 HARVONI®: Regional Sales Forecast, 2025-2030
Figure 9.13 Distribution by Type of Porphyria, 2021
Figure 9.14 Distribution by Type of Porphyria, 2030
Figure 9.15 Acute Hepatic Porphyria: Global Sales Forecast, 2021-2030
Figure 9.16 Acute Hepatic Porphyria: Regional Sales Forecast, 2021-2030
Figure 9.17 AIP: Global Sales Forecast, 2021-2030
Figure 9.18 AIP: Regional Sales Forecast, 2021-2030
Figure 9.19 EPP: Global Sales Forecast, 2025-2030
Figure 9.20 EPP: Regional Sales Forecast, 2021-2030
Figure 9.21 HCP: Global Sales Forecast, 2027-2030
Figure 9.22 HCP: Regional Sales Forecast, 2027-2030
Figure 9.23 PCT: Global Sales Forecast, 2025-2030
Figure 9.24 PCT: Regional Sales Forecast, 2025-2030
Figure 9.25 Variegate Porphyria: Global Sales Forecast, 2027-2030
Figure 9.26 Variegate Porphyria: Regional Sales Forecast, 2027-2030
Figure 9.27 X-Linked Porphyria: Global Sales Forecast, 2024-2030
Figure 9.28 X-Linked Porphyria: Regional Sales Forecast, 2024-2030
Figure 9.29 Distribution by Route of Administration, 2021
Figure 9.30 Distribution by Route of Administration, 2030
Figure 9.31 Oral: Global Sales Forecast, 2022-2030
Figure 9.32 Oral: Regional Sales Forecast, 2022-2030
Figure 9.33 Intravenous: Global Sales Forecast, 2021-2030
Figure 9.34 Intravenous: Regional Sales Forecast, 2021-2030
Figure 9.35 Subcutaneous: Global Sales Forecast, 2021-2030
Figure 9.36 Subcutaneous: Regional Sales Forecast, 2021-2030
Figure 9.37 Overall Market Opportunity, 2021-2030
Figure 9.38 Distribution by Type of Drug Class, 2021, 2025 and 2030
Figure 9.39 Regional Distribution, 2021,2025 and 2030
Figure 9.40 Country-wise Distribution (Bubble Chart Representation)
Companies Mentioned
- Access Biotechnology
- Agios Pharmaceuticals
- Alexion Pharmaceuticals
- Alnylam Pharmaceuticals
- Association pour la recherche en medecine interne (armi)
- Atlas Molecular Pharma
- Atlas Ventures
- Atrium Health Wake Forest Baptist
- Beam Therapeutics
- Biotech Lab Singapore
- Bioprofarma Bago
- Blackstone
- Brigham and Women's Hospital
- Celnova Pharma
- Chiesi Farmaceutici
- Clinuvel Pharmaceuticals
- Committee for Medicinal Products of Human Use (CHMP)
- Cycle Pharmaceuticals
- Das Nationale Centrum für Tumorerkrankungen (NCT)
- Digna Biotech
- Disc Medicine
- FARMOVS
- Forge Biologics
- Gilead Sciences
- Hong Kong Winhealth Pharma Group
- Hospital Universitario 12 de Octubre
- Icahn School of Medicine
- Ironwood Pharmaceuticals
- Italchimici
- Lundbeck
- Medison Pharma
- Mitsubishi Tanabe Pharma
- Moderna Therapeutics
- Mount Sinai Morningside
- National Center for Research Resources (NCRR)
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- National Institutes of Health (NIH)
- NeuroDerm
- Nordlandssykehuset
- Novartis
- Novo Holdings
- Office of Rare Diseases (ORD)
- Palatin Technologies
- PANTHERx Rare
- PAREXEL
- Recordati Rare Diseases
- Roche
- Strasbourg University Hospital
- SurModics
- Taiba
- The Royal Norwegian Ministry of Health
- The University of Navarra
- The University of Texas
- The University of Texas Medical Branch
- Therapeutic Goods Administration (TGA)
- uniQure
- University of Alabama
- University of California
- University of South Florida
- University of Tromsø
- University of Utah
Methodology
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