This “Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2021,” report provides comprehensive insights about 5+ companies and 5+ 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.
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. Mutations in the ATP8B1, ABCB11, and ABCB4 genes can cause PFIC. These mutations in different hepato-canalicular transporters lead to intrahepatic cholestasis (bile accumulation in the liver). Cellular bile accumulation leads to hepatocyte death.
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. A liver biopsy may be done to check the liver tissue. A highly specialized test measuring bile salt levels may be done to specifically check for PFIC. Genetic testing can reveal exactly mutated gene and provide information about the severity of the disease.
Treatment for Progressive Familial Intrahepatic Cholestasis (PFIC) is medical therapy, surgery and liver transplant. Ursodiol (also called ursodeoxycholic acid or UDCA) reduces elevated liver enzymes by easing bile flow through the liver. Rifampin (also called rifampicin) can be used to treat pruritus. Vitamin supplements can be taken in order to replenish the vitamins A, D, E, and K that are not being properly absorbed by the body through the bile. Liver transplantation may be necessary if liver failure or liver cirrhosis occurs.
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.
Maralixibat: Mirum Pharmaceuticals
Maralixibat is a novel, minimally absorbed, orally administered investigational drug. It inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, thereby potentially reducing bile acid mediated liver damage and related effects and complications. The FDA has granted Maralixibat Breakthrough Therapy designation for treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2.
VTX-803: Vivet Therapeutics
VTX-801 is a novel investigational gene therapy. It uses a novel modified AAV vector to transport a truncated functional version of the gene into the liver cells carrying the defective gene to treat the underlying cause of the disease. VTX-803 is currently under preclinical studies for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3). In June 2021, FDA and the European Commission (EC) have granted Orphan Drug Designation for VTX-803, for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3).
There are approx. 5+ key companies which are developing the therapies for Progressive Familial Intrahepatic Cholestasis (PFIC). The companies which have their Progressive Familial Intrahepatic Cholestasis (PFIC) drug candidates in the most advanced stage, i. e. phase III include, Mirum Pharmaceuticals.
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 II, I, preclinical and discovery stage. It also analyses Progressive Familial Intrahepatic Cholestasis (PFIC) 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 Progressive Familial Intrahepatic Cholestasis (PFIC) drugs.
Geography Covered
- Global coverage
Progressive Familial Intrahepatic Cholestasis (PFIC) Understanding
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. Mutations in the ATP8B1, ABCB11, and ABCB4 genes can cause PFIC. These mutations in different hepato-canalicular transporters lead to intrahepatic cholestasis (bile accumulation in the liver). Cellular bile accumulation leads to hepatocyte death.
Symptoms
Signs and symptoms of PFIC typically begin in infancy and are related to bile buildup and liver disease. 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
- Liver cancer
Diagnosis
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. A liver biopsy may be done to check the liver tissue. A highly specialized test measuring bile salt levels may be done to specifically check for PFIC. Genetic testing can reveal exactly mutated gene and provide information about the severity of the disease.
Treatment
Treatment for Progressive Familial Intrahepatic Cholestasis (PFIC) is medical therapy, surgery and liver transplant. Ursodiol (also called ursodeoxycholic acid or UDCA) reduces elevated liver enzymes by easing bile flow through the liver. Rifampin (also called rifampicin) can be used to treat pruritus. Vitamin supplements can be taken in order to replenish the vitamins A, D, E, and K that are not being properly absorbed by the body through the bile. Liver transplantation may be necessary if liver failure or liver cirrhosis occurs.
Progressive Familial Intrahepatic Cholestasis (PFIC) Emerging Drugs Chapters
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
Maralixibat: Mirum Pharmaceuticals
Maralixibat is a novel, minimally absorbed, orally administered investigational drug. It inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, thereby potentially reducing bile acid mediated liver damage and related effects and complications. The FDA has granted Maralixibat Breakthrough Therapy designation for treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2.
VTX-803: Vivet Therapeutics
VTX-801 is a novel investigational gene therapy. It uses a novel modified AAV vector to transport a truncated functional version of the gene into the liver cells carrying the defective gene to treat the underlying cause of the disease. VTX-803 is currently under preclinical studies for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3). In June 2021, FDA and the European Commission (EC) have granted Orphan Drug Designation for VTX-803, for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3).
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)
There are approx. 5+ key companies which are developing the therapies for Progressive Familial Intrahepatic Cholestasis (PFIC). The companies which have their Progressive Familial Intrahepatic Cholestasis (PFIC) drug candidates in the most advanced stage, i. e. phase III include, Mirum Pharmaceuticals.
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
Progressive Familial Intrahepatic Cholestasis (PFIC) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- intravitreal
- Subretinal
- Topical.
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Progressive Familial Intrahepatic Cholestasis (PFIC): Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase 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.
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).
- In December 2021, Mirum Pharmaceuticals and Oberland Capital Management announced a $200 million capped, tiered, revenue-based funding agreement based on net revenues of maralixibat, as well as a $10 million equity investment agreement. The funding from Oberland Capital will enable Mirum Pharmaceuticals to invest in the launch and commercialization of maralixibat in the United States and Europe.
Progressive Familial Intrahepatic Cholestasis (PFIC) Report Insights
- Progressive Familial Intrahepatic Cholestasis (PFIC) Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Progressive Familial Intrahepatic Cholestasis (PFIC) Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
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?
Key Players
- Mirum Pharmaceuticals
- Albireo
- Vivet Therapeutics
Key Products
- Maralixibat
- Odevixibat
- VTX-803
Table of Contents
IntroductionExecutive 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) - Analytical Perspective
In-depth Commercial Assessment
- Progressive Familial Intrahepatic Cholestasis (PFIC) companies’ collaborations, Licensing, Acquisition - Deal Value Trends
Progressive Familial Intrahepatic Cholestasis (PFIC) Collaboration Deals
- Company-Company Collaborations (Licensing / Partnering) Analysis
- Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
Maralixibat: Mirum Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Pre-clinical and Discovery Stage Products
- Comparative Analysis
VTX-803: Vivet Therapeutics
- Product Description
- Research and Development
- Product Development Activities
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
Progressive Familial Intrahepatic Cholestasis (PFIC) Key Companies
AppendixList of Tables
Table 1 Total Products for Progressive Familial Intrahepatic Cholestasis (PFIC)
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 Progressive Familial Intrahepatic Cholestasis (PFIC)
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
A selection of companies mentioned in this report includes:
- Mirum Pharmaceuticals
- Albireo
- Vivet Therapeutics
