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Propionic Acidemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034

May 2024 | 137 pages | ID: P88342694CE0EN
IMARC Group

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The 7 major propionic acidemia markets are expected to exhibit a CAGR of 2.74% during 2024-2034.

The propionic acidemia market has been comprehensively analyzed in IMARC's new report titled "Propionic Acidemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Propionic acidemia refers to a rare autosomal recessive metabolic disorder characterized by the impaired breakdown of certain amino acids and fatty acids in the body. This condition occurs due to a deficiency in the enzyme propionyl-CoA carboxylase, which is crucial for the catabolism of these substances. The symptoms of the ailment typically manifest during infancy or early childhood and can vary in severity. Individuals suffering from this illness may experience poor feeding, vomiting, lethargy, developmental delays, and metabolic crises that may lead to life-threatening complications, such as seizures and coma. Additionally, a distinctive sweet odor resembling that of maple syrup might emanate from the patient's bodily fluids. The diagnosis of this condition mainly involves analyzing blood and urine samples to detect elevated levels of propionic acid and related metabolites. The healthcare provider might also recommend molecular testing to confirm the presence of genetic mutations responsible for the enzyme deficiency.

The escalating incidences of genetic variations in the PCCA and PCCB genes, leading to the deficiency of an enzyme required for the breakdown of proteins and fats in the body, are primarily driving the propionic acidemia market. In addition to this, the inflating utilization of effective treatments, such as dietary management, which involves restricting the intake of certain amino acids and providing special formulas, along with carnitine supplementation to assist in metabolizing fatty acids, is also creating a positive outlook for the market. Moreover, the widespread adoption of awareness campaigns and early newborn screening programs that assist in timely detection and intervention is further bolstering the market growth. Apart from this, the rising usage of medical foods fortified with essential nutrients and vitamins, tailored to fulfill the specific nutritional needs of individuals suffering from propionic acidemia, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of physical and occupational therapies, since they aid in enhancing muscle tone, refining motor skills, and promoting overall physical development in patients, is also augmenting the market growth. Furthermore, the increasing demand for innovative approaches like gene therapy, which works by introducing functional genetic material to rectify or replace the mutated genes, is expected to drive the propionic acidemia market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the propionic acidemia 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 propionic acidemia 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 propionic acidemia 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 propionic acidemia market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the propionic acidemia market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current propionic acidemia 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 propionic acidemia 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 propionic acidemia market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the propionic acidemia 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 propionic acidemia across the seven major markets?
What is the number of prevalent cases (2018-2034) of propionic acidemia by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of propionic acidemia by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with propionic acidemia across the seven major markets?
What is the size of the propionic acidemia 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 propionic acidemia?
What will be the growth rate of patients across the seven major markets?

Propionic Acidemia: 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 propionic acidemia 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 propionic acidemia market?
What are the key regulatory events related to the propionic acidemia market?
What is the structure of clinical trial landscape by status related to the propionic acidemia market?
What is the structure of clinical trial landscape by phase related to the propionic acidemia market?
What is the structure of clinical trial landscape by route of administration related to the propionic acidemia market?
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 PROPIONIC ACIDEMIA - 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 PROPIONIC ACIDEMIA - 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 PROPIONIC ACIDEMIA - 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 PROPIONIC ACIDEMIA - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES

8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 PROPIONIC ACIDEMIA - UNMET NEEDS

10 PROPIONIC ACIDEMIA - KEY ENDPOINTS OF TREATMENT

11 PROPIONIC ACIDEMIA - MARKETED PRODUCTS

11.1 List of Propionic Acidemia Marketed Drugs Across the Top 7 Markets
  11.1.1 Drug Name – Company Name
    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 complete list of marketed drugs has been provided in the report.

12 PROPIONIC ACIDEMIA - PIPELINE DRUGS

12.1 List of Propionic Acidemia Pipeline Drugs Across the Top 7 Markets
  12.1.1 mRNA3927 - Moderna Therapeutics
    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
  12.1.2 HST5040 - HemoShear Therapeutics
    12.1.2.1 Drug Overview
    12.1.2.2 Mechanism of Action
    12.1.2.3 Clinical Trial Results
    12.1.2.4 Safety and Efficacy
    12.1.2.5 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 PROPIONIC ACIDEMIA - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS

14 PROPIONIC ACIDEMIA – 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 PROPIONIC ACIDEMIA - MARKET SCENARIO

15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
  15.2.1 Propionic Acidemia - Market Size
    15.2.1.1 Market Size (2018-2023)
    15.2.1.2 Market Forecast (2024-2034)
  15.2.2 Propionic Acidemia - 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 Propionic Acidemia - Market Size
    15.3.1.1 Market Size (2018-2023)
    15.3.1.2 Market Forecast (2024-2034)
  15.3.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview
15.4 Market Scenario - Germany
  15.4.1 Propionic Acidemia - Market Size
    15.4.1.1 Market Size (2018-2023)
    15.4.1.2 Market Forecast (2024-2034)
  15.4.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview
15.5 Market Scenario - France
  15.5.1 Propionic Acidemia - Market Size
    15.5.1.1 Market Size (2018-2023)
    15.5.1.2 Market Forecast (2024-2034)
  15.5.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
  15.6.1 Propionic Acidemia - Market Size
    15.6.1.1 Market Size (2018-2023)
    15.6.1.2 Market Forecast (2024-2034)
  15.6.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview
15.7 Market Scenario - Italy
  15.7.1 Propionic Acidemia - Market Size
    15.7.1.1 Market Size (2018-2023)
    15.7.1.2 Market Forecast (2024-2034)
  15.7.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview
15.8 Market Scenario - Spain
  15.8.1 Propionic Acidemia - Market Size
    15.8.1.1 Market Size (2018-2023)
    15.8.1.2 Market Forecast (2024-2034)
  15.8.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview
15.9 Market Scenario - Japan
  15.9.1 Propionic Acidemia - Market Size
    15.9.1.1 Market Size (2018-2023)
    15.9.1.2 Market Forecast (2024-2034)
  15.9.2 Propionic Acidemia - 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 Propionic Acidemia - Access and Reimbursement Overview

16 PROPIONIC ACIDEMIA - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS

17 PROPIONIC ACIDEMIA MARKET - SWOT ANALYSIS

17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 PROPIONIC ACIDEMIA MARKET – STRATEGIC RECOMMENDATIONS

19 APPENDIX


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