
Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2024
Description
Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2024
DelveInsight’s, “Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2024,” report provides comprehensive insights about 2+ companies and 3+ pipeline drugs in Progressive Familial Intrahepatic Cholestasis (PFIC) 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
Progressive Familial Intrahepatic Cholestasis (PFIC): Overview
Progressive Familial Intrahepatic Cholestasis (PFIC) is a rare genetic disorder that causes progressive, life-threatening liver disease, which typically leads to liver failure. The disease is inherited in an autosomal recessive pattern. In patients with PFIC, liver cells have a diminished ability to secrete bile due to mutations in proteins that control bile flow. The symptoms of Progressive Familial Intrahepatic Cholestasis (PFIC) include Pruritus (itching), yellowing of the skin and whites of the eyes (jaundice), failure to gain weight (failure to thrive), portal hypertension, enlarged liver and spleen (hepatosplenomegaly), cirrhosis, and liver cancer. A diagnosis of Progressive Familial Intrahepatic Cholestasis (PFIC) is made based upon a liver function tests, imaging test and /or biopsy. A liver function tests, are blood tests used to check the state of the liver or biliary system. Ultrasound, CT scan or MRI may be done to check the liver or biliary system. Treatment for Progressive Familial Intrahepatic Cholestasis (PFIC) is medical therapy, surgery and liver transplant.
""Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Progressive Familial Intrahepatic Cholestasis (PFIC) pipeline landscape is provided which includes the disease overview and Progressive Familial Intrahepatic Cholestasis (PFIC) treatment guidelines. The assessment part of the report embraces, in depth Progressive Familial Intrahepatic Cholestasis (PFIC) 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, Progressive Familial Intrahepatic Cholestasis (PFIC) 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 Progressive Familial Intrahepatic Cholestasis (PFIC) R&D. The therapies under development are focused on novel approaches to treat/improve Progressive Familial Intrahepatic Cholestasis (PFIC).
This segment of the Progressive Familial Intrahepatic Cholestasis (PFIC) 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.
Progressive Familial Intrahepatic Cholestasis (PFIC) Emerging Drugs
- VTX-803: Vivet Therapeutics
- Maralixibat: Mirum Pharmaceuticals
Further product details are provided in the report……..
Progressive Familial Intrahepatic Cholestasis (PFIC): Therapeutic Assessment
This segment of the report provides insights about the different Progressive Familial Intrahepatic Cholestasis (PFIC) drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Progressive Familial Intrahepatic Cholestasis (PFIC)
- 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
Progressive Familial Intrahepatic Cholestasis (PFIC): Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Progressive Familial Intrahepatic Cholestasis (PFIC) 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 Progressive Familial Intrahepatic Cholestasis (PFIC) drugs.
Progressive Familial Intrahepatic Cholestasis (PFIC) Report Insights
- Progressive Familial Intrahepatic Cholestasis (PFIC) 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 Progressive Familial Intrahepatic Cholestasis (PFIC) drugs?
- How many Progressive Familial Intrahepatic Cholestasis (PFIC) drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Progressive Familial Intrahepatic Cholestasis (PFIC) 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 Progressive Familial Intrahepatic Cholestasis (PFIC) and their status?
- What are the key designations that have been granted to the emerging drugs?
- Mirum Pharmaceuticals
- Vivet Therapeutics
- Maralixibat
- VTX-803
- VTX-802
Table of Contents
40 Pages
- Introduction
- Executive Summary
- Progressive Familial Intrahepatic Cholestasis (PFIC): 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
- Progressive Familial Intrahepatic Cholestasis (PFIC) – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Maralixibat: Mirum Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical/Discovery Stage Products
- Comparative Analysis
- VTX-803: Vivet Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Progressive Familial Intrahepatic Cholestasis (PFIC) Key Companies
- Progressive Familial Intrahepatic Cholestasis (PFIC) Key Products
- Progressive Familial Intrahepatic Cholestasis (PFIC)- Unmet Needs
- Progressive Familial Intrahepatic Cholestasis (PFIC)- Market Drivers and Barriers
- Progressive Familial Intrahepatic Cholestasis (PFIC)- Future Perspectives and Conclusion
- Progressive Familial Intrahepatic Cholestasis (PFIC) Analyst Views
- Appendix
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