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Polycystic Kidney Disease Drug Pipeline Analysis Report 2025

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    Report

  • 200 Pages
  • June 2025
  • Region: Global
  • Expert Market Research
  • ID: 6102716
According to the National Institute of Health, autosomal dominant polycystic kidney disease (ADPKD) affects 1 in every 400 to 1,000 people globally. Polycystic kidney disease is one of the most common genetic disorders and affects approximately 500,000 people in the United States. There has been an increasing need for an improved therapy for ADPKD. Major pharma companies and research institutes are developing innovative treatments to manage the disease leading to an increased number of drugs in the pipeline.

Report Coverage

The Polycystic Kidney Disease Drug Pipeline Insight Report by the publisher gives comprehensive insights into polycystic kidney disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for polycystic kidney disease. The polycystic kidney disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The polycystic kidney disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from the condition, and alignment with polycystic kidney disease treatment guidelines to ensure optimal care practices.

The detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration and ongoing polycystic kidney disease development activities are covered. Moreover, polycystic kidney disease collaborations and commercial assessments are offered, helping stakeholders to make informed decisions.

Polycystic Kidney Disease Drug Pipeline Outlook

Polycystic kidney disease is an inherited disorder, wherein clusters of cysts develop primarily within the kidneys, causing its enlargement and loss of function over time. These cysts are noncancerous round sacs containing fluid, that can vary in size, and grow very large. Polycystic kidney disease can also cause cysts to develop in the liver and other areas of the body. This can cause serious complications such as high blood pressure and kidney failure. According to the National Kidney Foundation, approximately 600,000 people have PKD in the United States and it is the fourth leading cause of kidney failure and causes about 5% of all kidney failures. The symptoms of polycystic kidney disease include high blood pressure, blood in urine, feeling of fullness in the abdomen, back or side pain, headaches, increased size of the abdomen due to enlarged kidneys, kidney stones and failure and urinary tract or kidney infections among others. However, the symptoms can vary, depending on the stage of the disease.

The are two main types of polycystic kidney disease, including autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD). The signs and symptoms of ADPKD often develop between the ages of 30 to 40 but children can also develop the disorder. If a single parent has ADPKD, each child has a 50% chance of getting the disease. ADPKD is the most common form of polycystic kidney disease while ARPKD is less common. The signs and symptoms often appear shortly after birth.

There is no cure for PKD currently. However, several research is being done to develop effective treatment alternatives. In April 2018, the U.S. FDA tolvaptan for autosomal dominant polycystic kidney disease treatment. This drug is used to slow down the decline of kidney function in adults at risk. Several pharma companies are developing innovative treatments to manage polycystic kidney disease. There has been a significant increase in the clinical trials for polycystic kidney disease. For instance, Otsuka is studying tolvaptan in two phase 3 studies in ARPKD, each evaluating the safety and effectiveness of tolvaptan in patients under the age of 18. Positive results from clinical trials are expected to impact the pipeline landscape significantly in the coming years.

Polycystic Kidney Disease - Drug Pipeline Therapeutic Assessment

This section of the report covers the analysis of polycystic kidney disease drug candidates based on several segmentations including:

By Phase

The pipeline assessment report covers 50+ drug analyses based on phase.
  • Late-Stage Products (Phase 3 and Phase 4)
  • Mid-Stage Products (Phase 2)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

By Drug Class

The polycystic kidney disease therapeutics assessment report covers 50+ drug analyses based on drug classes:

  • Recombinant Fusion Proteins
  • Small Molecule
  • Monoclonal Antibody
  • Peptide
  • Polymer
  • Gene Therapy
  • Kinase Inhibitor

By Route of Administration

The pipeline assessment report covers 50+ drug analyses based on the route of administration.
  • Oral
  • Parenteral
  • Others

Polycystic Kidney Disease - Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to analysis, phase II covers a major share of the total clinical trials for polycystic kidney disease with 44 pipeline drugs in phase II.

Polycystic Kidney Disease - Pipeline Assessment Segmentation, By Drug Classes

The drug molecules categories covered under polycystic kidney disease pipeline analysis include recombinant fusion proteins, small molecules, monoclonal antibodies, peptides, polymer, and gene therapy. A. Manzoni Hospital is conducting a phase II study to evaluate whether the administration of everolimus (a kinase inhibitor) slows down the progression of disease in ADPKD patients. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for polycystic kidney disease.

Polycystic Kidney Disease Clinical Trials Assessment - Competitive Dynamics

The polycystic kidney disease drug report insights involve the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in polycystic kidney disease clinical trials:
  • Palladio Biosciences
  • Regulus Therapeutics Inc.
  • Novartis Pharmaceuticals
  • Kadmon Corporation, LLC
  • Pfizer
  • Otsuka Pharmaceutical Development & Commercialization, Inc.
  • Rege Nephro Co., Ltd.
  • AceLink Therapeutics, Inc.
  • Vertex Pharmaceuticals Incorporated
  • Advice Pharma Group srl
  • Others

Polycystic Kidney Disease - Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases including phase I, phase II, phase III, phase IV, and emerging drugs for polycystic kidney disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of polycystic kidney disease drug candidates.

Drug: RGLS8429

The trial is designed to evaluate the safety and tolerability of RGLS8429 and to assess the impact of RGLS8429 on ADPKD biomarkers. The trial is sponsored by Regulus Therapeutics Inc. and is currently under phase II.

Drug: tolvaptan

The objective of the study is to assess the clinical safety and effectiveness of tolvaptan twice daily in ADPKD patients. The trial is sponsored by Otsuka Pharmaceutical Co., Ltd. and is currently under phase III.

Drug: AL01211

AceLink Therapeutics, Inc. is developing the drug and is currently under phase I. The study is being designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of Oral AL01211 in healthy volunteers.

Reasons To Buy This Report

The Polycystic Kidney Disease Drug Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for polycystic kidney disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into polycystic kidney disease collaborations, regulatory environments, and potential growth opportunities within the treatment landscape.

Key Questions Answered in the Polycystic Kidney Disease - Pipeline Assessment Report

  • Which companies/institutions are leading the polycystic kidney disease drug development?
  • What is the efficacy and safety profile of polycystic kidney disease pipeline drugs?
  • Which company is leading the polycystic kidney disease pipeline development activities?
  • What is the current polycystic kidney disease commercial assessment?
  • What are the opportunities and challenges present in the polycystic kidney disease drug pipeline landscape?
  • What is the efficacy and safety profile of polycystic kidney disease pipeline drugs?
  • Which company is conducting major trials for polycystic kidney disease drugs?
  • Which companies/institutions are involved in polycystic kidney disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
  • What geographies are covered for clinical trials in polycystic kidney disease?

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Table of Contents

1 Preface
1.1 Introduction
1.2 Objectives of the Study
1.3 Research Methodology and Assumptions
2 Executive Summary
3 Overview of Polycystic Kidney Disease
3.1 Signs and Symptoms
3.2 Causes
3.3 Risk Factors
3.4 Types of Polycystic Kidney Disease
3.5 Diagnosis
3.6 Treatment
4 Patient Profile: Polycystic Kidney Disease
4.1 Patient Profile Overview
4.2 Patient Psychology and Emotional Impact Factors
4.3 Risk Assessment and Treatment Success Rate
5 Polycystic Kidney Disease: Epidemiology Snapshot
5.1 Polycystic Kidney Disease Incidence by Key Markets
5.2 Polycystic Kidney Disease - Patients Seeking Treatment in Key Markets
6 Polycystic Kidney Disease: Market Dynamics
6.1 Market Drivers and Constraints
6.2 SWOT Analysis
7 Polycystic Kidney Disease: Key Facts Covered
7.1 Top Countries Contributing to Clinical Trials in Asia-Pacific
7.2 Top Countries Contributing to Clinical Trials in Europe
7.3 Top Countries Contributing to Clinical Trials in North America
7.4 Top Countries Contributing to Clinical Trials in Other Regions
8 Polycystic Kidney Disease, Drug Pipeline Assessment
8.1 Assessment by Treatment Type
8.2 Assessment by Route of Administration
8.3 Assessment by Drug Class
9 Drug Pipeline Comparative Analysis
9.1 List of Polycystic Kidney Disease Pipeline Drugs
9.1.1 By Company
9.1.2 By Phase
9.1.3 By Indication
9.1.4 By Trial Status
9.1.5 By Funder Type
9.2 Attribute Scoring Analysis of Pipeline Drugs (Top Drugs)
10 Polycystic Kidney Disease Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
10.1 Comparative Analysis for Late-Stage Drugs
10.1.1 Study Type
10.1.2 Recruitment Status
10.1.3 Company
10.1.4 Funder Type
10.2 Product Level Analysis*
10.2.1 Lixivaptan
10.2.1.1 Product Description
10.2.1.2 Trial ID
10.2.1.3 Sponsor Name
10.2.1.4 Study Type
10.2.1.5 Drug Class
10.2.1.6 Eligibility Criteria
10.2.1.7 Study Record Dates
10.2.1.7.1 First Submitted
10.2.1.7.2 First Posted
10.2.1.7.3 Last Update Posted
10.2.1.7.4 Last Verified
10.2.1.8 Indication
10.2.1.9 Study Design
10.2.1.10 Recruitment Status
10.2.1.11 Enrollment (Estimated)
10.2.1.12 Location Countries
10.2.1.13 Recent Results
10.2.2 Tolvaptan
10.2.3 Sirolimus
10.2.4 Other Drugs
11 Polycystic Kidney Disease Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
11.1 Comparative Analysis for Mid-Stage Drugs
11.1.1 Study Type
11.1.2 Recruitment Status
11.1.3 Company
11.1.4 Funder Type
11.2 Product Level Analysis*
11.2.1 Rapamune
11.2.1.1 Product Description
11.2.1.2 Trial ID
11.2.1.3 Sponsor Name
11.2.1.4 Study Type
11.2.1.5 Drug Class
11.2.1.6 Eligibility Criteria
11.2.1.7 Study Record Dates
11.2.1.7.1 First Submitted
11.2.1.7.2 First Posted
11.2.1.7.3 Last Update Posted
11.2.1.7.4 Last Verified
11.2.1.8 Indication
11.2.1.9 Study Design
11.2.1.10 Recruitment Status
11.2.1.11 Enrollment (Estimated)
11.2.1.12 Location Countries
11.2.1.13 Recent Results
11.2.2 Bosutinib
11.2.3 Other Drugs
12 Polycystic Kidney Disease Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
12.1 Comparative Analysis for Early-Stage Drugs
12.1.1 Study Type
12.1.2 Recruitment Status
12.1.3 Company
12.1.4 Funder Type
12.2 Product Level Analysis*
12.2.1 Tesevatinib
12.2.1.1 Product Description
12.2.1.2 Trial ID
12.2.1.3 Sponsor Name
12.2.1.4 Study Type
12.2.1.5 Drug Class
12.2.1.6 Eligibility Criteria
12.2.1.7 Study Record Dates
12.2.1.7.1 First Submitted
12.2.1.7.2 First Posted
12.2.1.7.3 Last Update Posted
12.2.1.7.4 Last Verified
12.2.1.8 Indication
12.2.1.9 Study Design
12.2.1.10 Recruitment Status
12.2.1.11 Enrollment (Estimated)
12.2.1.12 Location Countries
12.2.2 Midazolam
12.2.3 Other Drugs
13 Polycystic Kidney Disease Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
13.1 Comparative Analysis for Preclinical and Discovery Stage Drugs
13.1.1 Study Type
13.1.2 Recruitment Status
13.1.3 Company
13.1.4 Funder Type
13.2 Product Level Analysis*
13.2.1 Drug 1
13.2.1.1 Product Description
13.2.1.2 Trial ID
13.2.1.3 Sponsor Name
13.2.1.4 Study Type
13.2.1.5 Drug Class
13.2.1.6 Eligibility Criteria
13.2.1.7 Study Record Dates
13.2.1.7.1 First Submitted
13.2.1.7.2 First Posted
13.2.1.7.3 Last Update Posted
13.2.1.7.4 Last Verified
13.2.1.8 Indication
13.2.1.9 Study Design
13.2.1.10 Recruitment Status
13.2.1.11 Enrollment (Estimated)
13.2.1.12 Location Countries
13.2.2 Other Drugs
14 Polycystic Kidney Disease, Key Drug Pipeline Companies
14.1 Palladio Biosciences
14.1.1 Company Snapshot
14.1.2 Pipeline Product Portfolio
14.1.3 Financial Analysis
14.1.4 Recent News and Developments
14.2 Regulus Therapeutics Inc.
14.2.1 Company Snapshot
14.2.2 Pipeline Product Portfolio
14.2.3 Financial Analysis
14.2.4 Recent News and Developments
14.3 Novartis Pharmaceuticals
14.3.1 Company Snapshot
14.3.2 Pipeline Product Portfolio
14.3.3 Financial Analysis
14.3.4 Recent News and Developments
14.4 Kadmon Corporation, LLC
14.4.1 Company Snapshot
14.4.2 Pipeline Product Portfolio
14.4.3 Financial Analysis
14.4.4 Recent News and Developments
14.5 Pfizer
14.5.1 Company Snapshot
14.5.2 Pipeline Product Portfolio
14.5.3 Financial Analysis
14.5.4 Recent News and Developments
14.6 Otsuka Pharmaceutical Development & Commercialization, Inc.
14.6.1 Company Snapshot
14.6.2 Pipeline Product Portfolio
14.6.3 Financial Analysis
14.6.4 Recent News and Developments
14.7 Rege Nephro Co., Ltd.
14.7.1 Company Snapshot
14.7.2 Pipeline Product Portfolio
14.7.3 Financial Analysis
14.7.4 Recent News and Developments
14.8 AceLink Therapeutics, Inc.
14.8.1 Company Snapshot
14.8.2 Pipeline Product Portfolio
14.8.3 Financial Analysis
14.8.4 Recent News and Developments
14.9 Vertex Pharmaceuticals Incorporated
14.9.1 Company Snapshot
14.9.2 Pipeline Product Portfolio
14.9.3 Financial Analysis
14.9.4 Recent News and Developments
14.10 Advice Pharma Group srl
14.10.1 Company Snapshot
14.10.2 Pipeline Product Portfolio
14.10.3 Financial Analysis
14.10.4 Recent News and Developments
15 Regulatory Framework for Drug Approval, By Region16 Terminated or Suspended Pipeline Products