Summary

The 7 major pyruvate kinase deficiency markets are expected to exhibit a CAGR of 3.99% during 2024-2034.

The pyruvate kinase deficiency market has been comprehensively analyzed in IMARC's new report titled "Pyruvate Kinase Deficiency Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Pyruvate kinase deficiency refers to a genetic disorder that affects the red blood cells (erythrocytes) in the body. It is caused by changes (mutations) in the PKLR gene, resulting in a lack of the enzyme pyruvate kinase. People with this illness are often affected by chronic hemolytic anemia, a condition in which red blood cells prematurely undergo hemolysis, causing their shortage in the body. Indications of pyruvate kinase deficiency might vary significantly from person to person. Some of the common symptoms include an enlarged spleen, shortness of breath, yellowish skin, dark-colored urine, high concentrations of iron, weakness, etc. The diagnosis of this disease typically involves a full patient and family history, a thorough clinical evaluation, and a series of specialized tests. Furthermore, the medical practitioner may perform lab tests to determine the presence and cause of anemia. A low hemoglobin and haptoglobin level, along with an elevated unconjugated bilirubin level and reticulocyte count in the blood, are all indicative of confirmed disease in patients.

The rising prevalence of inherited genetic conditions on account of gene abnormalities, mutations in the DNA, and chromosomal irregularities is primarily driving the pyruvate kinase deficiency market. Furthermore, the widespread adoption of folic acid supplements to produce new red blood cells and reduce the risk of complications associated with the disorder is also augmenting the market growth. Apart from this, the inflating utilization of phototherapy in infants with pyruvate kinase deficiency, since it lowers the total serum bilirubin concentration without any likelihood of neurotoxicity, is further creating a positive outlook for the market. Additionally, several key players are investing in R&D activities for the launch of novel disease-modifying gene therapies, which allow the replacement of a mutated gene with a healthy one. This, in turn, is acting as another significant growth-inducing factor. Moreover, the increasing demand for an allosteric pyruvate kinase activator that works by upregulating the activity of erythrocyte pyruvate kinase and enhancing the lifespan of red blood cells in the body is expected to drive the pyruvate kinase deficiency market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the pyruvate kinase deficiency market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for pyruvate kinase deficiency and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the pyruvate kinase deficiency market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the pyruvate kinase deficiency market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the pyruvate kinase deficiency market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current pyruvate kinase deficiency marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the pyruvate kinase deficiency market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the pyruvate kinase deficiency market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the pyruvate kinase deficiency market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of pyruvate kinase deficiency across the seven major markets?
What is the number of prevalent cases (2018-2034) of pyruvate kinase deficiency by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of pyruvate kinase deficiency by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with pyruvate kinase deficiency across the seven major markets?
What is the size of the pyruvate kinase deficiency patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of pyruvate kinase deficiency?
What will be the growth rate of patients across the seven major markets?

Pyruvate Kinase Deficiency: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for pyruvate kinase deficiency drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the pyruvate kinase deficiency market?
What are the key regulatory events related to the pyruvate kinase deficiency market?
What is the structure of clinical trial landscape by status related to the pyruvate kinase deficiency market?
What is the structure of clinical trial landscape by phase related to the pyruvate kinase deficiency market?
What is the structure of clinical trial landscape by route of administration related to the pyruvate kinase deficiency market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Pyruvate Kinase Deficiency - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Pyruvate Kinase Deficiency - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Pyruvate Kinase Deficiency - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)

8 Pyruvate Kinase Deficiency - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Pyruvate Kinase Deficiency - Unmet Needs

10 Pyruvate Kinase Deficiency - Key Endpoints of Treatment

11 Pyruvate Kinase Deficiency - Marketed Products
11.1 List of Pyruvate Kinase Deficiency Marketed Drugs Across the Top 7 Markets
11.1.1 Pyrukynd (Mitapivat) - Agios Pharmaceuticals
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets

Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 Pyruvate Kinase Deficiency - Pipeline Drugs
12.1 List of Pyruvate Kinase Deficiency Pipeline Drugs Across the Top 7 Markets
12.1.1 RPL301 - Rocket Pharmaceuticals
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Pyruvate Kinase Deficiency - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Pyruvate Kinase Deficiency – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Pyruvate Kinase Deficiency - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Pyruvate Kinase Deficiency - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Pyruvate Kinase Deficiency - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Pyruvate Kinase Deficiency - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Pyruvate Kinase Deficiency - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Pyruvate Kinase Deficiency - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Pyruvate Kinase Deficiency - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Pyruvate Kinase Deficiency - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Pyruvate Kinase Deficiency - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Pyruvate Kinase Deficiency - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Pyruvate Kinase Deficiency - Access and Reimbursement Overview

16 Pyruvate Kinase Deficiency - Recent Events and Inputs From Key Opinion Leaders

17 Pyruvate Kinase Deficiency Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Pyruvate Kinase Deficiency Market – Strategic Recommendations

19 Appendix