Atypical Hemolytic Uremic Syndrome (aHUS) - Pipeline Insight, 2024

This “Atypical Hemolytic Uremic Syndrome (a HUS) - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Atypical Hemolytic Uremic Syndrome (a HUS) 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 inthisspace.

Geography Covered

• Global coverage

Atypical Hemolytic Uremic Syndrome (a HUS)Understanding

Atypical Hemolytic Uremic Syndrome (a HUS):Overview

Atypical hemolytic uremic syndrome (a HUS) is a thrombotic microangiopathy (TMA) that affects multiple organs and the kidneys in particular. a HUS can be sporadic or familial and is most commonly caused by dysregulation of the alternative complement pathway. The initial attack of aHUS can occur at any age, and is associated with a high rate of progression to end stage renal disease. The majority of aHUS is caused by complement system defects impairing ordinary regulatory mechanisms. Activating events therefore lead to unbridled, ongoing complement activity producing widespread endothelial injury. Pathologic mutations include those resulting in loss-of-function in a complement regulatory gene (CFH, CFI, CD46 or THBD) or gain-of-function in an effector gene (CFB or C3). Treatment with the late complement inhibitor, eculizumab - a monoclonal antibody directed against C5 -iseffective.

Atypical Hemolytic Uremic Syndrome (a HUS) - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Atypical Hemolytic Uremic Syndrome (a HUS) pipeline landscape is provided which includes the disease overview and Atypical Hemolytic Uremic Syndrome (aHUS) treatment guidelines. The assessment part of the report embraces, in depth Atypical Hemolytic Uremic Syndrome (aHUS) 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, collaborations, licensing, mergers and acquisition, funding, designations and other productrelateddetails.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence in Atypical Hemolytic Uremic Syndrome (a HUS) R&D. The therapies under development are focused on novel approaches to treat/improve in Atypical Hemolytic Uremic Syndrome(aHUS).

This segment of the Atypical Hemolytic Uremic Syndrome (a HUS) report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and pressreleases.

Atypical Hemolytic Uremic Syndrome (a HUS) EmergingDrugs

Iptacopan: Novartis Pharmaceuticals Iptacopan is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH. In doing so, iptacopan may have a therapeutic advantage over anti-C5 therapies by targeting a key part of the biology responsible for PNH while offering an oral monotherapy option. Discovered at the Novartis Institutes for Bio Medical Research, iptacopan is currently in development for a number of other complement-mediated diseases (CMDs) where significant unmet needs exist, including kidney diseases C3G, Ig AN, atypical hemolytic uremic syndrome (a HUS), membranous nephropathy (MN), lupus nephritis (LN), and blood disorders immune thrombocytopenic purpura (ITP) and coldagglutinindisease(CAD).

Atypical Hemolytic Uremic Syndrome (a HUS): TherapeuticAssessment

This segment of the report provides insights about the different Atypical Hemolytic Uremic Syndrome (a HUS) drugs segregated based on following parameters that define the scope of the report, suchas:

Major Players in Atypical Hemolytic Uremic Syndrome (aHUS)

There are approx. 5+ key companies which are developing the therapies for Atypical Hemolytic Uremic Syndrome (a HUS). The companies which have their Atypical Hemolytic Uremic Syndrome (a HUS) drug candidates in the most advanced stage, i.e. Phase III include,NovartisPharmaceuticals.

Phases

This report covers around 5+ products under different phases of clinical development like

• 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

Atypical Hemolytic Uremic Syndrome (a HUS) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs suchas

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Atypical Hemolytic Uremic Syndrome (a HUS): Pipeline DevelopmentActivities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Atypical Hemolytic Uremic Syndrome (a HUS) therapeutic drugs key players involved in developing keydrugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Atypical Hemolytic Uremic Syndrome (a HUS)drugs.

Atypical Hemolytic Uremic Syndrome (a HUS) ReportInsights

• Atypical Hemolytic Uremic Syndrome (a HUS) PipelineAnalysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Atypical Hemolytic Uremic Syndrome (a HUS) ReportAssessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Atypical Hemolytic Uremic Syndrome (a HUS)drugs?
• How many Atypical Hemolytic Uremic Syndrome (a HUS) drugs are developed by eachcompany?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Atypical Hemolytic Uremic Syndrome (aHUS)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Atypical Hemolytic Uremic Syndrome (aHUS)?
• 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 Hemolytic Uremic Syndrome (a HUS) and theirstatus?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Novartis Pharmaceuticals
• Chugai Pharmaceutical
• Tasly Biopharmaceuticals
• Prestige BioPharma

Key Products

• Iptacopan
• Crovalimab
• B 2067 2
• Eculizumab

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