Progressive Familial Intrahepatic Cholestasis Pipeline: Promising Therapies Shaping the Future of PFIC Treatment | DelveInsight

The treatment landscape for Progressive Familial Intrahepatic Cholestasis (PFIC), a rare and progressive liver disorder, is evolving rapidly, driven by innovative therapies from leading pharmaceutical and biotech companies. Key players such as Mirum Pharmaceuticals and Vivet Therapeutics are advancing therapeutic options focused on addressing the root causes of cholestasis, including bile acid regulation and hepatic transport. These emerging treatments aim to improve liver function, reduce pruritus, and delay the progression of liver damage in patients suffering from PFIC.

DelveInsight's "Progressive Familial Intrahepatic Cholestasis - Pipeline Insight, 2025" provides an in-depth analysis of the current clinical development landscape, featuring investigational therapies across multiple stages, from early preclinical research to late-stage clinical trials. The report examines the mechanisms of action of each drug, their development status, and expected timelines for market entry.

This pipeline insight offers a comprehensive 360° view of PFIC therapeutic trends, including drug types, routes of administration, and stages of clinical development. Additionally, it highlights unmet needs, strategic collaborations, regulatory designations, and emerging market trends expected to reshape the treatment paradigm for PFIC in the years ahead.

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Key Takeaways from the Progressive Familial Intrahepatic Cholestasis Pipeline Report
• DelveInsight's progressive familial intrahepatic cholestasis pipeline analysis depicts a strong space with 2+ active players working to develop 3+ pipeline drugs for progressive familial intrahepatic cholestasis treatment.
• The leading progressive familial intrahepatic cholestasis companies include Mirum Pharmaceuticals, Vivet Therapeutics, and others are evaluating their lead assets to improve the progressive familial intrahepatic cholestasis treatment landscape.
• Key progressive familial intrahepatic cholestasis pipeline therapies in various stages of development include Maralixibat, VTX-803, VTX-802, and others.
• In April 2025, Mirum Pharmaceuticals (Nasdaq: MIRM) announced FDA approval for a new tablet formulation of LIVMARLI® (maralixibat) to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
• In August 2024, Innorna announced that the FDA granted Orphan Drug Designation (ODD) to its investigational therapy, IN016, for treating Progressive Familial Intrahepatic Cholestasis (PFIC). IN016 also received Rare Pediatric Disease Designation (RPDD) in July 2024, accelerating its clinical development and potential approval.

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Progressive Familial Intrahepatic Cholestasis Overview
Progressive Familial Intrahepatic Cholestasis (PFIC) is a rare genetic condition that causes progressive liver damage, often leading to liver failure. The disorder is inherited in an autosomal recessive manner and occurs due to mutations in proteins responsible for bile secretion in the liver. Common symptoms include intense itching (pruritus), jaundice, poor weight gain, portal hypertension, hepatosplenomegaly, cirrhosis, and liver cancer. Diagnosis is typically made through liver function tests, imaging (ultrasound, CT scan, or MRI), and sometimes liver biopsy. Treatment options include medical management, surgery, and liver transplantation.

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Progressive Familial Intrahepatic Cholestasis Treatment Analysis: Drug Profile
VTX-803: Vivet Therapeutics
VTX-803 is an innovative gene therapy currently in preclinical development for treating Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3). It utilizes a modified AAV vector to deliver a truncated, functional version of the defective gene directly into liver cells. In June 2020, both the FDA and the European Commission (EC) granted Orphan Drug Designation to VTX-803 for PFIC3 treatment.

Maralixibat: Mirum Pharmaceuticals
Maralixibat is a novel, minimally absorbed oral investigational drug that targets the apical sodium-dependent bile acid transporter (ASBT). By inhibiting ASBT, it promotes the excretion of bile acids through feces, potentially reducing bile acid-induced liver damage and associated complications. The FDA has granted Maralixibat Breakthrough Therapy Designation for the treatment of pruritus in patients with Alagille Syndrome (ALGS) aged one year and older, as well as for PFIC2.

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Progressive Familial Intrahepatic Cholestasis Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• 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

By Route of Administration
• Oral
• Parenteral
• Intravitreal
• Subretinal
• Topical

By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Progressive Familial Intrahepatic Cholestasis Pipeline Report
• Coverage: Global
• Key Progressive Familial Intrahepatic Cholestasis Companies: Mirum Pharmaceuticals, Vivet Therapeutics, and others.
• Key Progressive Familial Intrahepatic Cholestasis Pipeline Therapies: Maralixibat, VTX-803, VTX-802, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Progressive Familial Intrahepatic Cholestasis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Progressive Familial Intrahepatic Cholestasis Pipeline Therapeutics
6. Progressive Familial Intrahepatic Cholestasis Pipeline: Late-Stage Products (Phase III)
7. Progressive Familial Intrahepatic Cholestasis Pipeline: Mid-Stage Products (Phase II)
8. Progressive Familial Intrahepatic Cholestasis Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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This release was published on openPR.