PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Outlook and Forecast

PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Published Date : 2022-07-19

Updated On : 2023-05-01

Pages : 154

PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Outlook

Thelansis’s “PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential PKP2-Arrhythmogenic Cardiomyopathy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Overview

Arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) is a hereditary cardiac disorder characterized by impaired function of the right ventricle (RV) and the occurrence of ventricular arrhythmias, which can ultimately lead to sudden cardiac death. The condition is characterized by the replacement of cardiomyocytes with fibrofatty tissue. ARVD/C is typically inherited in an autosomal dominant manner, but it exhibits incomplete penetrance, and the expression of the disease varies significantly with age. In rare cases, autosomal recessive inheritance can occur with or without cutaneous involvement. The progressive fibrotic replacement process leads to electrical instability, increasing the risk of syncope and sudden cardiac death, or causing alterations in cardiac function, resembling dilated cardiomyopathy, which may result in right- or bi-ventricular heart failure. The diagnosis of ARVD/C is challenging and relies on applying the 2010 Task Force criteria, which consider family history, distinctive electrocardiographic findings, arrhythmic events, and structural and histological abnormalities. Mutations in the PKP2 gene are responsible for 9-43% of ARVC cases and are considered the most common genetic cause of the condition. However, it should be noted that a single pathogenic variant in PKP2 may not be sufficient to cause the full manifestation of the disease. The PKP2 gene encodes a protein that belongs to the Armadillo (ARM) repeat family. Plakophilin proteins, including Plakophilin-2, contain multiple ARM repeats and are localized in cell desmosomes and nuclei. They play a crucial role in linking cadherins to intermediate filaments in the cytoskeleton. Plakophilin-2 is essential for the assembly of junctional proteins and serves as a vital morphogenic factor and architectural component in the heart. ARVC often exhibits familial clustering, with a family history of the condition present in 30-50% of cases.

  • Additionally, men tend to have a higher incidence and greater severity of ARVC than women. ARVC has an estimated prevalence of 1 per 5000 individuals in the general population.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2022-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Competitive Landscape

S. no Asset Company Stage
1 RP-A601 Rocket Pharmaceuticals Phase 1/2

KOLs across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

COUNTRY No. Of KOLs
USA 17
GERMANY 4
UK 4
SPAIN 3
FRANCE 2
ITALY 3
JAPAN 3
CHINA 4

PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast

1.       PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) – Key Findings Summary

         1.1.    Clinical findings
                     1.1.1. Disease overview
                     1.1.2. Therapeutic practices
                     1.1.3. Future outlook
         1.2.    Commercial findings
                     1.2.1. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) market scenario 2022
                     1.2.2. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) market scenario 2025
                     1.2.3. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) market scenario 2032

2.    PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Overview

         2.1.    Disease Introduction
         2.2.    Pathophysiology
         2.3.    Signs and Symptoms
         2.4.    Risk Factors
         2.5.    Etiology
         2.6.    Classification
         2.7.    Pathogenesis
         2.8.    Diagnosis
         2.9.    Complications
         2.10. Treatment Algorithm
                     2.10.1.    Treatment in US (guidelines)
                     2.10.2.    Treatment in EU-5 (guidelines)
                     2.10.3.    Treatment in Japan (guidelines)
                     2.10.4.    Treatment in China (guidelines)
         2.11. Treatment Goals for PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
         2.12. Referral Patterns
                     2.12.1.    Referral Scenario in US
                     2.12.2.    Referral Scenario in EU-5
                     2.12.3.    Referral Scenario in Japan
                     2.12.4.    Referral Scenario in China
         2.13. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Prognosis
         2.14. Healthcare burden
                     2.14.1.    Healthcare burden in US
                     2.14.2.    Healthcare burden in EU-5
                     2.14.3.    Healthcare burden in Japan
                     2.14.4.    Healthcare burden in China
         2.15.  Unmet Needs in PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) management
         2.16.  Market Opportunity for PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
         2.17. KOL Comments on current and upcoming/expected treatment practices in PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Epidemiology in US (2022-2032)
                             3.2.1.1.              Incidence of PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Epidemiology in EU-5 (2022-2032)
                             3.2.2.1.              Incidence of PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Epidemiology in Japan (2022-2032)
                             3.2.3.1.              Incidence of PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4. PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Epidemiology in China (2022-2032)
                             3.2.4.1.              Incidence of PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
                             3.2.4.2.              Diagnosed cases
                             3.2.4.3.              Treatable Patient Pool
                             3.2.4.4.              Epidemiology Trends
         3.3.    Epidemiology Trends (World-wide)

4.       Market Outlook

         4.1.    US PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.1.1. Market Progression (Futuristic)
                     4.1.2. Market Trends and Expectations
                             4.1.2.1.              Worst case scenario
                             4.1.2.2.              Base Case Scenario
                             4.1.2.3.              Best Case Scenario
                     4.1.3. Drivers and Barriers
         4.2.    UK PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.2.1. Market Progression (Futuristic)
                     4.2.2. Market Trends and Expectations
                             4.2.2.1.              Worst case scenario
                             4.2.2.2.              Base Case Scenario
                             4.2.2.3.              Best Case Scenario
                     4.2.3. Drivers and Barriers
         4.3.    France PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.3.1. Market Progression (Futuristic)
                     4.3.2. Market Trends and Expectations
                             4.3.2.1.              Worst case scenario
                             4.3.2.2.              Base Case Scenario
                             4.3.2.3.              Best Case Scenario
                     4.3.3. Drivers and Barriers
         4.4.    Germany PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.4.1. Market Progression (Futuristic)
                     4.4.2. Market Trends and Expectations
                             4.4.2.1.              Worst case scenario
                             4.4.2.2.              Base Case Scenario
                             4.4.2.3.              Best Case Scenario
                     4.4.3. Drivers and Barriers
         4.5.    Italy PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.5.1. Market Progression (Futuristic)
                     4.5.2. Market Trends and Expectations
                             4.5.2.1.              Worst case scenario
                             4.5.2.2.              Base Case Scenario
                             4.5.2.3.              Best Case Scenario
                     4.5.3. Drivers and Barriers
         4.6.    Spain PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.6.1. Market Progression (Futuristic)
                     4.6.2. Market Trends and Expectations
                             4.6.2.1.              Worst case scenario
                             4.6.2.2.              Base Case Scenario
                             4.6.2.3.              Best Case Scenario
                     4.6.3. Drivers and Barriers
         4.7.    Japan PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.7.1. Market Progression (Futuristic)
                     4.7.2. Market Trends and Expectations
                             4.7.2.1.              Worst case scenario
                             4.7.2.2.              Base Case Scenario
                             4.7.2.3.              Best Case Scenario
                     4.7.3. Drivers and Barriers
         4.8.    China PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market Forecast 2022-2032
                     4.8.1. Market Progression (Futuristic)
                     4.8.2. Market Trends and Expectations
                             4.8.2.1.              Worst case scenario
                             4.8.2.2.              Base Case Scenario
                             4.8.2.3.              Best Case Scenario
                     4.8.3. Drivers and Barriers
         4.9.    Key Expected Milestones (world-wide) Impacting the Market

5.       Competitive Landscape

         5.1.    Pipeline Therapies Overview
                     5.1.1. Phase III Therapies
                                      5.1.1.1.                     Current Status
                                      5.1.1.2.                     Trial details, results
                                      5.1.1.3.                     Approval Timeline
                                      5.1.1.4.                     Likelihood of approval
                                      5.1.1.5.                     Expected Product Positioning
                             5.1.1.2.              All other Phase III Therapies …..
                             5.1.1.3.              Attribute Analysis of Phase III molecules
                     5.1.2.  Phase II and Phase I/II Therapies
                                      5.1.2.1.                     Current Status
                                      5.1.2.2.                     Trial details, results
                                      5.1.2.3.                     Approval Timelines
                     5.1.3. List of active Pre-clinical Therapies
                                      5.1.3.1.                     Status in PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
                                      5.1.3.2.                     Company positioning
                             5.1.3.2.              All other pre-clinical therapies
                     5.1.4. List of Inactive/discontinued assets
                             5.1.4.1.              Business impact of discontinuations on current pipeline
                     5.1.5. Potential winners from PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Pipeline
                             5.1.5.1.              Potential Blockbusters across the pipeline

6.       Regulatory/Approval Scenario

         6.1.    Regulatory/Approval Framework in US
                     6.1.1. Policy Framework
                     6.1.2. Payer Expectations
         6.2.    Regulatory/Approval Framework in UK
                     6.2.1. Policy Framework
                     6.2.2. Payer Expectations
         6.3.    Regulatory/Approval Framework in France
                     6.3.1. Policy Framework
                     6.3.2. Payer Expectations
         6.4.    Regulatory/Approval Framework in Germany
                     6.4.1. Policy Framework
                     6.4.2. Payer Expectations
         6.5.    Regulatory/Approval Framework in Italy
                     6.5.1. Policy Framework
                     6.5.2. Payer Expectations
         6.6.    Regulatory/Approval Framework in Spain
                     6.6.1. Policy Framework
                     6.6.2. Payer Expectations
         6.7.       Regulatory/Approval Framework in Japan
                     6.7.1. Policy Framework
                     6.7.2. Payer Expectations
         6.8.       Regulatory/Approval Framework in China
                     6.8.1. Policy Framework
                     6.8.2. Payer Expectations

7.       Clinical Trial Assessment – Current and Future Paradigm

         7.1.    Distribution of Primary Endpoints across trials
         7.2.    Distribution of Secondary Endpoints across trials
         7.3.    Evolution and acceptance of surrogate endpoints
         7.4.    Key Investigator initiated trials
         7.5.    Attrition analysis
                     7.5.1. Suspended/Discontinued Assets
                     7.5.2. Failed Trials, Reasons and Business Impact
                     7.5.3. Terminated Trials, Reasons and Business Impact
                     7.5.4. Withdrawn Trials, Reasons and Business Impact
         7.6.    Trial enrollment scenario and challenges
         7.7.    Clinical Trial Guidance (across geographies)

8.       Thelansis Commentary

         8.1.    Key Unmet needs in PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
         8.2.    Possible Best-case Clinical Trial Strategies
         8.3.    Possible Best Case Targeted Product Profile (TPP)
         8.4.    Possible Best-case Market positioning strategies
         8.5.    Possible Best-case Market Access Strategies
         8.6.    Possible Best-case LCM Strategies
         8.7.    Overall View on PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM) Market in Dollar Value

9.       Report Methodology

         9.1.    Secondary research
         9.2.    Primary research
         9.3.    Data collation
         9.4.    Insight Generation

10.   About Thelansis

         10.1.    Our Capabilities
         10.2.    Our Services
         10.3.    Our Contacts
         10.4.    Disclaimer