Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2025

This “Progressive Familial Intrahepatic Cholestasis (PFIC) - Pipeline Insight, 2025,” 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.

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. 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, 2025' report 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).

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

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 2020, FDA and the European Commission (EC) had granted Orphan Drug Designation for VTX-803, for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3).

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.

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. 2+ 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

The report covers around 3+ 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

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 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

Progressive Familial Intrahepatic Cholestasis (PFIC) Report Assessment

• 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 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
• Vivet Therapeutics

Key Products

• Maralixibat
• VTX-803
• VTX-802