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Global Porphyria Pipeline Report 2021-2031: Clinical Trials, KOL Analysis, Company Competitiveness, Publication Analysis & Partnership and Collaborations

·6-min read

Dublin, Nov. 15, 2021 (GLOBE NEWSWIRE) -- The "Porphyria: Pipeline Review, Developer Landscape and Competitive Insights, 2021-2031" report has been added to's offering.

This 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.

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.

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?

Key Topics Covered:

1.1. Context
1.2. Project Report



4.1. Market Landscape of Porphyria Drugs
4.2. Market Landscape of Porphyria Drug Developers

5.1 Case Study on Terminated and Dormant Programs

6.1. Clinical Trial Analysis
6.2. KOL Analysis
6.3. Company Competitiveness Analysis
6.4. Publication Analysis
6.6 Partnership and Collaborations

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

8.1. Drugs Forecasted and Sales Estimated
8.1.2. PanhematinT
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

9.1. 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

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 fur Tumorerkrankungen (NCT)

  • Digna Biotech

  • Disc Medicine


  • 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


  • 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 Tromso

  • University of Utah

For more information about this report visit

CONTACT: CONTACT: Laura Wood, Senior Press Manager For E.S.T Office Hours Call 1-917-300-0470 For U.S./CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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