Summary

The 7 major homocystinuria markets are expected to exhibit a CAGR of 3% during 2024-2034.

The homocystinuria market has been comprehensively analyzed in IMARC's new report titled "Homocystinuria Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Homocystinuria is a rare genetic disorder that mainly affects the ability of the body to break down certain amino acids, specifically methionine. The most common type of homocystinuria is caused by a deficiency of an enzyme called cystathionine beta-synthase (CBS). This can lead to excess formation of homocysteine and other related substances in the blood and urine. Symptoms of homocystinuria can vary widely and may include developmental delays, intellectual disability, seizures, visual problems, chest deformities, abnormal bone development, blood clot formation, etc. In some cases, patients may also experience failure to gain weight at an expected rate, movement problems, spindly arms and legs, dislocation of the lens at the front of the eye, seizures, nearsightedness, etc. The diagnosis of homocystinuria is typically based on the patient's medical history, indications, and physical exam. A blood test is also utilized to measure elevated levels of amino acids in the body. The healthcare provider may further perform various studies, including genetic testing, liver biopsies, hepatic enzyme tests, etc., to validate a disease diagnosis among patients.

The rising cases of genetic disorders, causing gene mutations that can prevent the normal functioning of the enzymes, are primarily driving the homocystinuria market. Besides this, the widespread adoption of vitamin B6 and folic acid supplements, which can decrease blood levels of homocysteine and provide symptom relief, is also augmenting the market growth. Furthermore, the escalating application of lifestyle modifications, such as consuming a low-methionine diet, avoiding alcohol, practicing aerobic and strength-training exercises, etc., to slow the disease progression and enhance the quality of life in patients, is creating a positive outlook for the market. Additionally, various key players are making significant investments in R&D activities to launch novel therapeutic formulations that can control elevated levels of amino acids in critical illnesses. This, in turn, is further bolstering the market growth. Moreover, the emerging popularity of PEGylated recombinant enzyme replacement therapy with pegtibatinase, which aims to introduce the CBS enzyme into circulation, thereby reducing homocysteine concentrations, is expected to drive the homocystinuria market in the coming years.

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

Homocystinuria: 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 homocystinuria 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 homocystinuria market?
What are the key regulatory events related to the homocystinuria market?
What is the structure of clinical trial landscape by status related to the homocystinuria market?
What is the structure of clinical trial landscape by phase related to the homocystinuria market?
What is the structure of clinical trial landscape by route of administration related to the homocystinuria 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 Homocystinuria - 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 Homocystinuria - 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 Homocystinuria - 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 Homocystinuria - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Homocystinuria - Unmet Needs

10 Homocystinuria - Key Endpoints of Treatment

11 Homocystinuria - Marketed Products
11.1 List of Homocystinuria Marketed Drugs Across the Top 7 Markets
11.1.1 Cystadane (Betaine) - Recordati
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 Homocystinuria - Pipeline Drugs
12.1 List of Homocystinuria Pipeline Drugs Across the Top 7 Markets
12.1.1 Pegtibatinase - Travere 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 Pegtarviliase - Aeglea Biotherapeutics
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
12.1.3 SYNB1353 - Synlogic
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 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. Homocystinuria - Attribute Analysis of Key Marketed and Pipeline Drugs

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

16 Homocystinuria - Recent Events and Inputs From Key Opinion Leaders

17 Homocystinuria Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Homocystinuria Market – Strategic Recommendations

19 Appendix