This “Atypical Haemolytic Uraemic Syndrome - Pipeline Insight, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Atypical Haemolytic Uraemic Syndrome 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.
Atypical hemolytic uremic syndrome (aHUS) is an extremely rare disease characterized by low levels of circulating red blood cells due to their destruction (hemolytic anemia), low platelet count (thrombocytopenia) due to their consumption and inability of the kidneys to process waste products from the blood and excrete them into the urine (acute kidney failure), a condition known as uremia. It is a distinctly different illness from the more common disorder known as typical hemolytic uremic syndrome, which is caused by E.coli-producing Shiga toxins (Stx HUS) and is generally foodborne. The onset of atypical hemolytic uremic syndrome ranges from before birth (prenatally) to adulthood. In young children, the disorder often develops suddenly and usually follows an infection, particularly an upper respiratory infection or gastroenteritis.
Most cases of aHUS are associated with mutations amongst the multiple genes that produce (encode) proteins involved in the alternate pathway of complement, which is part of the complement system of the innate immune system. Treatment by a medical team familiar with the unique challenges of aHUS is recommended and can include pediatricians or general internists, kidney specialists (nephrologists), intensive care physicians, nurses, nutritionists and social workers. Initially, affected individuals may receive supportive care including maintaining proper nutrition and electrolyte and fluid balance through intravenous feeding (parenteral) when and if necessary.
The companies and academics are working to assess challenges and seek opportunities that could influence Atypical Haemolytic Uraemic Syndrome R&D. The therapies under development are focused on novel approaches for Atypical Haemolytic Uraemic Syndrome.
This segment of the Atypical Haemolytic Uraemic Syndrome 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.
Crovalimab: Chugai Pharmaceutical/Roche
Crovalimab acts as inhibitors of Complement C5. It is being developed by Chugai Pharmaceutical/Roche for Atypical Haemolytic Uraemic Syndrome and is currently in phase III stage of development. It is administered intravenously.
rVA576: Akari Therapeutics
Nomacopan (rVA576) is a second-generation complement inhibitor which acts on complement component-C5, preventing release of C5a and formation of C5b-9 (also known as the membrane attack complex, or MAC), and independently also inhibits leukotriene B4, or LTB4, activity, both elements that are co-located as part of the immune/inflammatory response. Nomacopan is a recombinant small protein (16,740 Da) derived from a protein originally discovered in the saliva of the Ornithodoros moubata tick, where it modulates the host immune system to allow the parasite to feed without alerting the host to its presence or provoking an immune response. It is currently in phase III stage of development and is being developed by Akari Therapeutics.
There are approx. 3+ key companies which are developing the Atypical Haemolytic Uraemic Syndrome. The companies which have their Atypical Haemolytic Uraemic Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Chugai Pharmaceutical/Roche.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Atypical Haemolytic Uraemic Syndrome therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Atypical Haemolytic Uraemic Syndrome drugs.
Geography Covered
- Global coverage
Atypical Haemolytic Uraemic Syndrome Understanding
Atypical Haemolytic Uraemic Syndrome: Overview
Atypical hemolytic uremic syndrome (aHUS) is an extremely rare disease characterized by low levels of circulating red blood cells due to their destruction (hemolytic anemia), low platelet count (thrombocytopenia) due to their consumption and inability of the kidneys to process waste products from the blood and excrete them into the urine (acute kidney failure), a condition known as uremia. It is a distinctly different illness from the more common disorder known as typical hemolytic uremic syndrome, which is caused by E.coli-producing Shiga toxins (Stx HUS) and is generally foodborne. The onset of atypical hemolytic uremic syndrome ranges from before birth (prenatally) to adulthood. In young children, the disorder often develops suddenly and usually follows an infection, particularly an upper respiratory infection or gastroenteritis.
Most cases of aHUS are associated with mutations amongst the multiple genes that produce (encode) proteins involved in the alternate pathway of complement, which is part of the complement system of the innate immune system. Treatment by a medical team familiar with the unique challenges of aHUS is recommended and can include pediatricians or general internists, kidney specialists (nephrologists), intensive care physicians, nurses, nutritionists and social workers. Initially, affected individuals may receive supportive care including maintaining proper nutrition and electrolyte and fluid balance through intravenous feeding (parenteral) when and if necessary.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Atypical Haemolytic Uraemic Syndrome R&D. The therapies under development are focused on novel approaches for Atypical Haemolytic Uraemic Syndrome.
Atypical Haemolytic Uraemic Syndrome Emerging Drugs Chapters
This segment of the Atypical Haemolytic Uraemic Syndrome 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.
Atypical Haemolytic Uraemic Syndrome Emerging Drugs
Crovalimab: Chugai Pharmaceutical/Roche
Crovalimab acts as inhibitors of Complement C5. It is being developed by Chugai Pharmaceutical/Roche for Atypical Haemolytic Uraemic Syndrome and is currently in phase III stage of development. It is administered intravenously.
rVA576: Akari Therapeutics
Nomacopan (rVA576) is a second-generation complement inhibitor which acts on complement component-C5, preventing release of C5a and formation of C5b-9 (also known as the membrane attack complex, or MAC), and independently also inhibits leukotriene B4, or LTB4, activity, both elements that are co-located as part of the immune/inflammatory response. Nomacopan is a recombinant small protein (16,740 Da) derived from a protein originally discovered in the saliva of the Ornithodoros moubata tick, where it modulates the host immune system to allow the parasite to feed without alerting the host to its presence or provoking an immune response. It is currently in phase III stage of development and is being developed by Akari Therapeutics.
Atypical Haemolytic Uraemic Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Atypical Haemolytic Uraemic Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Major Players working on Atypical Haemolytic Uraemic Syndrome
There are approx. 3+ key companies which are developing the Atypical Haemolytic Uraemic Syndrome. The companies which have their Atypical Haemolytic Uraemic Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Chugai Pharmaceutical/Roche.
Phases
This report covers around 3+ products under different phases of clinical development like
- Late-stage products (Phase III and
- Mid-stage products (Phase II and
- Early-stage products (Phase I/II and Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Atypical Haemolytic Uraemic Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Intravaginal
- Oral
- Parenteral
- Subcutaneous
- Topical
- Molecule Type
Products have been categorized under various Molecule types such as
- Vaccines
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Atypical Haemolytic Uraemic Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Atypical Haemolytic Uraemic Syndrome 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 Atypical Haemolytic Uraemic Syndrome drugs.
Atypical Haemolytic Uraemic Syndrome Report Insights
- Atypical Haemolytic Uraemic Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Atypical Haemolytic Uraemic Syndrome Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Scenario and Emerging Therapies:
- How many companies are developing Atypical Haemolytic Uraemic Syndrome drugs?
- How many Atypical Haemolytic Uraemic Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for Atypical Haemolytic Uraemic Syndrome?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Atypical Haemolytic Uraemic Syndrome 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 Atypical Haemolytic Uraemic Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Chugai Pharmaceutical/Roche
- AKARI Therapeutics
- Prestige BioPharma
Key Products
- Crovalimab
- rVA576
- PBP 1603
Table of Contents
IntroductionExecutive Summary
Atypical Haemolytic Uraemic Syndrome: Overview
- Structure
- Mechanism of Action
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
Atypical Haemolytic Uraemic Syndrome- Analytical Perspective
In-depth Commercial Assessment
- Atypical Haemolytic Uraemic Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Atypical Haemolytic Uraemic Syndrome Collaboration Deals
- Company-Company Collaborations (Licensing/Partnering) Analysis
- Company-University Collaborations (Licensing/Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
Crovalimab: Chugai Pharmaceutical/Roche
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I)
- Comparative Analysis
PBP 1603: Prestige BioPharma
- Product Description
- Research and Development
- Product Development Activities
Preclinical and Discovery Stage Products
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Atypical Haemolytic Uraemic Syndrome Key Companies
Atypical Haemolytic Uraemic Syndrome Key Products
Atypical Haemolytic Uraemic Syndrome- Unmet Needs
Atypical Haemolytic Uraemic Syndrome- Market Drivers and Barriers
Atypical Haemolytic Uraemic Syndrome- Future Perspectives and Conclusion
Atypical Haemolytic Uraemic Syndrome Analyst Views
Atypical Haemolytic Uraemic Syndrome Key Companies
AppendixList of Tables
Table 1 Total Products for Atypical Haemolytic Uraemic Syndrome
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 ProductsList of Figures
Figure 1 Total Products for Atypical Haemolytic Uraemic Syndrome
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
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Chugai Pharmaceutical/Roche
- AKARI Therapeutics
- Prestige BioPharma
