Polycystic Kidney Disease Market

• Polycystic kidney disease (PKD) is a genetic disorder characterized by the uncontrolled growth of cysts in the kidneys, which can ultimately lead to kidney failure. There are of two types: autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD).
• Autosomal dominant polycystic kidney disease (ADPKD) is a prevalent cause of end-stage renal disease. This progressive and currently incurable condition leads to substantial morbidity and kidney failure.
• Imaging tests like abdominal ultrasound, CT scans, and MRI scans are used to confirm the presence of cysts in the kidneys and other organs. Blood and urine tests are also conducted to assess kidney function and look for signs of kidney damage. Genetic testing may be necessary for a definitive diagnosis in some cases
• Treatment aims to manage symptoms and prevent complications. Controlling high blood pressure is crucial. JYNARQUE (tolvaptan) is an approved drug that can slow the progression of cyst development in autosomal recessive polycystic kidney disease. Other treatments may include pain medication, blood pressure medication, antibiotics, and lifestyle modifications like a low-sodium diet and regular exercise.
• Key pharmaceutical companies like Rege Nephro, Regulus Therapeutics, Vertex Pharmaceuticals, and others are currently working on improving the treatment landscape of polycystic kidney disease. Recently, in March 2024, the FDA announced clearance of an Investigational New Drug Application for Vertex’s VX-407 for the treatment of autosomal dominant polycystic kidney.

DelveInsight's “Polycystic Kidney Disease Market Insights, Epidemiology and Market Forecast – 2034” report delivers an in-depth understanding of polycystic kidney disease, historical and forecasted epidemiology as well as the polycystic kidney disease market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

Polycystic Kidney Disease market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM polycystic kidney disease market size from 2020 to 2034. The report also covers current polycystic kidney disease treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Polycystic Kidney Disease Understanding and Treatment Algorithm

Polycystic Kidney Disease Overview, Country-Specific Treatment Guidelines and Diagnosis

Polycystic kidney disease is a genetic disorder that causes many cysts to grow in the kidneys. Polycystic kidney disease cysts cause high blood pressure and problems with blood vessels in the brain and heart. Cysts in the liver can also occur with polycystic kidney disease. There are two types of polycystic kidney disease: autosomal dominant polycystic kidney disease and autosomal recessive polycystic kidney disease.

Diagnosing polycystic kidney disease involves several tests and imaging exams to identify cysts in the kidneys. The most common and reliable method is ultrasound, a non-invasive procedure that uses sound waves to visualize kidney cysts. Additionally, CT scans and MRI scans can detect smaller cysts that might not be visible on ultrasound. Genetic testing may be recommended in certain cases, such as when the diagnosis is unclear based on imaging tests or if there is a family history of polycystic kidney disease.

The Polycystic Kidney Disease report provides an overview of polycystic kidney disease pathophysiology, diagnostic approaches, and detailed treatment algorithm along with a real-world scenario of a patient’s journey beginning from the first symptom, the time taken for diagnosis to the entire treatment process.

Further details related to country-based variations in diagnosis are provided in the report...

Polycystic Kidney Disease Treatment

There is currently no cure for polycystic kidney disease, but treatment focuses on managing symptoms, slowing the progression of the disease, and preventing complications. Medications may be prescribed to control high blood pressure, and pain management techniques can help alleviate discomfort. In advanced cases, kidney transplantation may be considered as a treatment option.

Polycystic Kidney Disease Epidemiology

The polycystic kidney disease epidemiology chapter in the report provides historical as well as forecasted in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. The polycystic kidney disease epidemiology is segmented with detailed insights into total diagnosed prevalent cases of polycystic kidney disease, Type-specific diagnosed prevalent cases of polycystic kidney disease, Age-specific diagnosed prevalent cases of polycystic kidney disease, Treatable cases of polycystic kidney disease.

• Polycystic Kidney Disease is the fourth leading cause of kidney failure in the US.
• Autosomal Recessive Polycystic Kidney Disease is a rare form of the disease that occurs in 1 in 25,000 children worldwide.
• According to the findings, autosomal recessive polycystic kidney disease affects males and females in equal numbers. The occurrence of autosomal recessive polycystic kidney disease is estimated to occur in approximately 1 in 20,000 individuals in the general population.
• Further, approximately 1/70 people carry a single mutation in the PKHD1 gene. As some people may go undiagnosed, it is difficult to determine the true frequency of ARPKD in the general population.

Polycystic Kidney Disease Drug Chapters

The drug chapter segment of the polycystic kidney disease report encloses a detailed analysis of polycystic kidney disease marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the polycystic kidney disease pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.

Marketed Drugs

JYNARQUE (tolvaptan): Otsuka Pharmaceutical

Tolvaptan is a selective vasopressin V2-receptor antagonist with an affinity for the V2-receptor that is 1.8 times that of native arginine vasopressin (AVP). Tolvaptan affinity for the V2-receptor is 29 times that for the V1areceptor. Decreased binding of vasopressin to the V2-receptor in the kidney lowers adenylate cyclase activity -cyclic monophosphate (cAMP) concentrations.

In April 2018, tolvaptan was approved by the US FDA under the brand name JYNARQUE. It was granted orphan-drug designation by the European Commission (EC) in August 2013 and received approval in May 2015 for the same condition, after receiving marketing authorization from the EMA, it was sold under the brand name JINARC. In March 2014, tolvaptan, received regulatory approval under the brand name SAMSCA, in Japan.

Note: Detailed current therapies assessment will be provided in the full report of Polycystic Kidney Disease...

Emerging Drugs

RN-014: Rege Nephro

Tamibarotene, a retinoic acid receptor (RAR) agonist, is being developed as a treatment for autosomal dominant polycystic kidney disease and has entered an early Phase II clinical trial for the targeted patients. The clinical trial is currently seeking participants in Japan.

Note: Detailed emerging therapies assessment will be provided in the final report...

RGLS8429: Regulus Therapeutics

RGLS8429 is a novel, next-generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney.   RGLS8429 had a favorable safety and PK profile was well-tolerated with no serious adverse events in Phase I the company is currently recruiting candidates in Phase Ib to evaluate RGLS8429 treatment across three different dose levels, including measuring changes in polycystins, height-adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function.

Polycystic Kidney Disease Market Outlook

Key players, such as Rege Nephro, Regulus Therapeutics, Vertex Pharmaceuticals, and others are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of polycystic kidney disease.

Polycystic Kidney Disease Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024–2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

Further detailed analysis of emerging therapies drug uptake in the report...

Polycystic Kidney Disease Activities

The report provides insights into different therapeutic candidates in Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for polycystic kidney disease emerging therapies.

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.

DelveInsight’s analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging treatment patterns of polycystic kidney disease. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial’s primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, descriptive overview of polycystic kidney disease, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression along with country-specific treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the polycystic kidney disease market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM polycystic kidney disease market.

Polycystic Kidney Disease Report Insights

• Patient Population
• Therapeutic Approaches
• Polycystic Kidney Disease Pipeline Analysis
• Polycystic Kidney Disease Market Size and Trends
• Existing and future Market Opportunity

Polycystic Kidney Disease Report Key Strengths

• Eleven Years Forecast
• 7MM Coverage
• Polycystic Kidney Disease Epidemiology Segmentation
• Inclusion of Country specific treatment guidelines
• KOL’s feedback on approved and emerging therapies
• Key Cross Competition
• Conjoint analysis
• Drugs Uptake and Key Market Forecast Assumptions

Polycystic Kidney Disease Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What is the growth rate of the 7MM polycystic kidney disease treatment market?
• What was the polycystic kidney disease total market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors/key catalysts for this growth?
• Is there any unexplored patient setting that can open the window for growth in the future?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the current and emerging options for the treatment of polycystic kidney disease?
• How many companies are developing therapies for the treatment of polycystic kidney disease?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• Patient/physician acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the polycystic kidney disease Market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.