Paroxysmal nocturnal haemoglobinuria - Pipeline Insight, 2021
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DelveInsight’s, “Paroxysmal nocturnal haemoglobinuria - Pipeline Insight, 2021,” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Paroxysmal nocturnal haemoglobinuria pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Paroxysmal nocturnal haemoglobinuria: Overview
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired, life-threatening disease of the blood. The disease is characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. PNH leads to excessive breakdown of red blood cells, leading to the release of a large amount of haemoglobin into the urine. Symptoms and signs of PNH include: fatigue; dark red/brown urine; difficulty swallowing, abdominal pain, infections, and bruising. The principal studies used to establish the diagnosis of PNH are flow cytometry of peripheral blood and bone marrow analysis. PNH typically starts from the early thirties to the mid?forties, and often persisting for decades, with a continued dependence on blood transfusions in a proportion of patients. The appropriate treatment for PNH depends on the severity of symptoms. The mainstay of PNH treatment is the drug eculizumab (Soliris).
'Paroxysmal nocturnal haemoglobinuria - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Paroxysmal nocturnal haemoglobinuria pipeline landscape is provided which includes the disease overview and Paroxysmal nocturnal haemoglobinuria treatment guidelines. The assessment part of the report embraces, in depth Paroxysmal nocturnal haemoglobinuria commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Paroxysmal nocturnal haemoglobinuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Paroxysmal nocturnal haemoglobinuria report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Paroxysmal nocturnal haemoglobinuria Emerging Drugs
Further product details are provided in the report
Paroxysmal nocturnal haemoglobinuria: Therapeutic Assessment
This segment of the report provides insights about the different Paroxysmal nocturnal haemoglobinuria drugs segregated based on following parameters that define the scope of the report, such as:
Paroxysmal nocturnal haemoglobinuria: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Paroxysmal nocturnal haemoglobinuria therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Paroxysmal nocturnal haemoglobinuria drugs.
Paroxysmal nocturnal haemoglobinuria Report Insights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Paroxysmal nocturnal haemoglobinuria - Pipeline Insight, 2021,” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Paroxysmal nocturnal haemoglobinuria pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Paroxysmal nocturnal haemoglobinuria: Overview
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired, life-threatening disease of the blood. The disease is characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. PNH leads to excessive breakdown of red blood cells, leading to the release of a large amount of haemoglobin into the urine. Symptoms and signs of PNH include: fatigue; dark red/brown urine; difficulty swallowing, abdominal pain, infections, and bruising. The principal studies used to establish the diagnosis of PNH are flow cytometry of peripheral blood and bone marrow analysis. PNH typically starts from the early thirties to the mid?forties, and often persisting for decades, with a continued dependence on blood transfusions in a proportion of patients. The appropriate treatment for PNH depends on the severity of symptoms. The mainstay of PNH treatment is the drug eculizumab (Soliris).
'Paroxysmal nocturnal haemoglobinuria - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Paroxysmal nocturnal haemoglobinuria pipeline landscape is provided which includes the disease overview and Paroxysmal nocturnal haemoglobinuria treatment guidelines. The assessment part of the report embraces, in depth Paroxysmal nocturnal haemoglobinuria commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Paroxysmal nocturnal haemoglobinuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Paroxysmal nocturnal haemoglobinuria R&D. The therapies under development are focused on novel approaches to treat/improve Paroxysmal nocturnal haemoglobinuria.
This segment of the Paroxysmal nocturnal haemoglobinuria report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Paroxysmal nocturnal haemoglobinuria Emerging Drugs
- Pegcetacoplan (APL-2): Apellis Pharmaceuticals
- Pozelimab: Regeneron Pharmaceuticals
Further product details are provided in the report
Paroxysmal nocturnal haemoglobinuria: Therapeutic Assessment
This segment of the report provides insights about the different Paroxysmal nocturnal haemoglobinuria drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Paroxysmal nocturnal haemoglobinuria
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Parenteral
- Intravitreal
- Subretinal
- Topical
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Paroxysmal nocturnal haemoglobinuria: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Paroxysmal nocturnal haemoglobinuria therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Paroxysmal nocturnal haemoglobinuria drugs.
Paroxysmal nocturnal haemoglobinuria Report Insights
- Paroxysmal nocturnal haemoglobinuria Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Paroxysmal nocturnal haemoglobinuria drugs?
- How many Paroxysmal nocturnal haemoglobinuria drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Paroxysmal nocturnal haemoglobinuria?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Paroxysmal nocturnal haemoglobinuria therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Paroxysmal nocturnal haemoglobinuria and their status?
- What are the key designations that have been granted to the emerging drugs?
- Alexion Pharmaceuticals
- Apellis Pharmaceuticals
- Regeneron Pharmaceuticals
- Akari Therapeutics
- Chugai Pharmaceutical
- Alnylam Pharmaceuticals
- BioCryst Pharmaceuticals
- Kira Pharmaceuticals
- Omeros Corporation
- Ra Pharmaceuticals
- Turgut Ilacari
- Vemircopan
- Pegcetacoplan (APL-2)
- Pozelimab
- Nomacopan
- Crovalimab
- Cemdisiran
- BCX-9930
- KP-104
- MASP-2 therapeutics
- RA 101295
- Research programme: biosimilar therapeutics
Introduction
Executive Summary
Paroxysmal nocturnal haemoglobinuria: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Paroxysmal nocturnal haemoglobinuria – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Paroxysmal nocturnal haemoglobinuria companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Paroxysmal nocturnal haemoglobinuria Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Preregistration)
Comparative Analysis
Pegcetacoplan: Apellis Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Late Stage Products (Phase III)
Comparative Analysis
Pozelimab: Regeneron Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Mid Stage Products (Phase II)
Comparative Analysis
Vemircopan: Alexion Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Preclinical and Discovery Stage Products
Comparative Analysis
KP-104: Kira Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Paroxysmal nocturnal haemoglobinuria Key Companies
Paroxysmal nocturnal haemoglobinuria Key Products
Paroxysmal nocturnal haemoglobinuria- Unmet Needs
Paroxysmal nocturnal haemoglobinuria- Market Drivers and Barriers
Paroxysmal nocturnal haemoglobinuria- Future Perspectives and Conclusion
Paroxysmal nocturnal haemoglobinuria Analyst Views
Paroxysmal nocturnal haemoglobinuria Key Companies
Appendix
Executive Summary
Paroxysmal nocturnal haemoglobinuria: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Paroxysmal nocturnal haemoglobinuria – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Paroxysmal nocturnal haemoglobinuria companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Paroxysmal nocturnal haemoglobinuria Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Preregistration)
Comparative Analysis
Pegcetacoplan: Apellis Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Late Stage Products (Phase III)
Comparative Analysis
Pozelimab: Regeneron Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Mid Stage Products (Phase II)
Comparative Analysis
Vemircopan: Alexion Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Preclinical and Discovery Stage Products
Comparative Analysis
KP-104: Kira Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Paroxysmal nocturnal haemoglobinuria Key Companies
Paroxysmal nocturnal haemoglobinuria Key Products
Paroxysmal nocturnal haemoglobinuria- Unmet Needs
Paroxysmal nocturnal haemoglobinuria- Market Drivers and Barriers
Paroxysmal nocturnal haemoglobinuria- Future Perspectives and Conclusion
Paroxysmal nocturnal haemoglobinuria Analyst Views
Paroxysmal nocturnal haemoglobinuria Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Paroxysmal nocturnal haemoglobinuria
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Paroxysmal nocturnal haemoglobinuria
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Paroxysmal nocturnal haemoglobinuria
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Paroxysmal nocturnal haemoglobinuria
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
