Summary

The 7 major Leber congenital amaurosis markets are expected to exhibit a CAGR of 3.23% during 2024-2034.

The Leber congenital amaurosis market has been comprehensively analyzed in IMARC's new report titled "Leber Congenital Amaurosis Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Leber congenital amaurosis (LCA) is a rare genetic disorder that primarily affects the retina and causes vision loss from birth or early childhood. The symptoms of LCA typically appear in the first few months of life and generally include progressive vision loss, nystagmus (involuntary eye movements), and photophobia (sensitivity to light). Some children with LCA may also experience a decline in visual acuity, the ability to distinguish fine details, and difficulty with visual tracking and eye coordination. The diagnosis of LCA usually entails a thorough eye examination, including visual acuity testing, visual field testing, and inspection of the retina and optic nerve. Additional tests, such as electroretinography (ERG), optical coherence tomography (OCT), and genetic testing, may also be performed to confirm the diagnosis and determine the underlying genetic cause. While OCT is a non-invasive imaging technique that produces fine cross-sectional images of the retina, ERG assesses the electrical activity of the retina in response to light stimulation.

The rising incidences of inherited eye diseases coupled with the inflating demand for treatments that can slow the progression of vision loss and improve quality of life are primarily driving the global Leber congenital amaurosis market. In addition to this, the widespread adoption of non-invasive imaging tests, such as optical coherence tomography, to provide detailed images of the retina and aid in diagnosing retinal diseases like LCA is also bolstering the market growth. Moreover, several key players are making extensive investments in order to gain an understanding of the genetic causes of LCA for the development of new therapies. This, in turn, is acting as another significant growth-inducing factor. Besides this, the increasing utilization of low-vision devices, including magnifiers, large print books, electronic devices, etc., to help individuals with LCA make the most of their remaining vision is also creating a positive outlook for the market. Furthermore, numerous government agencies and private organizations are offering educational and advocacy resources for LCA patients and their families, thereby propelling the market growth. Apart from this, the emerging popularity of occupational therapy and rehabilitation services among individuals with LCA for developing the skills and techniques they need to perform everyday activities, such as reading and navigating their environment, is expected to drive the global Leber congenital amaurosis market in the coming years.

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

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

9 Leber Congenital Amaurosis - Unmet Needs

10 Leber Congenital Amaurosis - Key Endpoints of Treatment

11 Leber Congenital Amaurosis - Marketed Products
11.1 List of Leber Congenital Amaurosis Marketed Drugs Across the Top 7 Markets
11.1.1 Luxturna (Voretigene neparvovec) - Spark Therapeutics
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 Leber Congenital Amaurosis - Pipeline Drugs
12.1 List of Leber Congenital Amaurosis Pipeline Drugs Across the Top 7 Markets
12.1.1 QR 110 - ProQR 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 SAR439483 - Atsena 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
12.1.3 EDIT101 - Editas Medicine
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. Leber Congenital Amaurosis - Attribute Analysis of Key Marketed and Pipeline Drugs

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

16 Leber Congenital Amaurosis - Recent Events and Inputs From Key Opinion Leaders

17 Leber Congenital Amaurosis Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Leber Congenital Amaurosis Market – Strategic Recommendations

19 Appendix