DelveInsight’s “Leber Congenital Amaurosis Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Leber Congenital Amaurosis market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Leber Congenital Amaurosis drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Leber Congenital Amaurosis treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Leber Congenital Amaurosis: An Overview
Leber congenital amaurosis (LCA) is a rare inherited eye disorder that causes severe vision loss at birth. The disease primarily affects the retina and is the most common cause of inherited blindness in childhood.
LCA is inherited in an autosomal recessive manner, meaning that both parents must carry a defective gene for the condition to pass it on to children. There is a 25% chance of inheriting the two LCA genes (one from each parent) needed to cause the disorder. Multiple genetic mutations have been identified to cause LCA. Therefore, new gene therapies for LCA are under development. Gene therapy is currently available for LCA caused by two mutations in a gene called RPE65, which accounts for about 6% of LCA cases.
Babies born with LCA have a very reduced vision that can often be detected by parents in the first few months of life. They may not respond to visual cues and have roving eye movements, called nystagmus. Many children with LCA habitually press their eyes with their fists or fingers, which causes their eyes to look sunken or deep-set.
In 2017, the gene therapy Luxturna (voretigene neparvovec-rzyl) was approved by the US Food and Drug Administration (FDA) to treat children and adults with two mutations in the RPE65 gene, which includes a type of LCA called LCA2. Luxturna is manufactured by Spark Therapeutics. Some of the major pharma and biotech giants such as ProQR Therapeutics, Atsena Therapeutics, and others are developing new therapies for Leber Congenital Amaurosis.
Leber Congenital Amaurosis Market Key Facts
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According to the National Organization for Rare Disorders, the prevalence of Leber congenital amaurosis (LCA) has been estimated to be 1-2 per 100,000 births.
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According to Coussa et al. (2017), LCA is the second most common group of inherited retinal dystrophies after retinitis pigmentosa, accounting for about 5% of all retinal dystrophies. With an estimated prevalence ranging from 1 per 33,000 to 1 per 81,000, LCA was reported to account for about 20% of legal blindness in children.
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Genetic and Rare Diseases Information Center (2021) estimated that the number of people with LCA in the US is between 3,000 and 30,000.
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As per the American Association for Pediatric Ophthalmology & Strabismus (2022), LCA is rare (2-3 cases per 100,000 births); about 10-18% of all cases of congenital blindness or severely reduced vision in children are caused by LCA.
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Leber congenital amaurosis market dynamics are anticipated to change in the coming years due to the development of emerging therapies. Key players such as ProQR Therapeutics, Atsena Therapeutics, and others are actively working in the Leber Congenital Amaurosis Market.
Leber Congenital Amaurosis Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Leber Congenital Amaurosis pipeline therapies. It also thoroughly assesses the Leber Congenital Amaurosis market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the market trend for each marketed Leber Congenital Amaurosis drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Leber Congenital Amaurosis Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Leber Congenital Amaurosis epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Leber Congenital Amaurosis epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Leber Congenital Amaurosis Epidemiology, Segmented as –
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Total Prevalent cases of Leber Congenital Amaurosis in the 7MM [2019-2032]
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Gene-specific cases of Leber Congenital Amaurosis in the 7MM [2019-2032]
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Total Diagnosed Prevalent cases of Leber Congenital Amaurosis in the 7MM [2019-2032]
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Treatable cases of Leber Congenital Amaurosis in the 7MM [2019-2032]
Leber Congenital Amaurosis Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Leber Congenital Amaurosis market or expected to be launched during the study period. The analysis covers the Leber Congenital Amaurosis market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Leber Congenital Amaurosis drugs based on their sale and market share.
The report also covers the Leber Congenital Amaurosis pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Leber Congenital Amaurosis companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Leber Congenital Amaurosis Therapeutics Analysis
Several major pharma and biotech companies are developing therapies for Leber Congenital Amaurosis. Currently, ProQR Therapeutics is leading the therapeutics market with its Leber Congenital Amaurosis drug candidates in the most advanced stage of clinical development.
Leber Congenital Amaurosis Companies Actively Working in the Therapeutics Market Include
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ProQR Therapeutics
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Editas Medicine
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Atsena Therapeutics Inc.
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Applied Genetic Technologies Corporation
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IVERIC bio
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MeiraGTx
And Many Others
Emerging and Marketed Leber Congenital Amaurosis Therapies Covered in the Report Include:
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Sepofarsen (QR-110): ProQR Therapeutics
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EDIT-101: Editas Medicine, Inc.
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Luxturna: Spark Therapeutics
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ATSN-101: Atsena Therapeutics
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Leber Congenital Amaurosis Competitive Intelligence Analysis
4. Leber Congenital Amaurosis Market Overview at a Glance
5. Leber Congenital Amaurosis Disease Background and Overview
6. Leber Congenital Amaurosis Patient Journey
7. Leber Congenital Amaurosis Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Leber Congenital Amaurosis Treatment Algorithm, Current Treatment, and Medical Practices
9. Leber Congenital Amaurosis Unmet Needs
10. Key Endpoints of Leber Congenital Amaurosis Treatment
11. Leber Congenital Amaurosis Marketed Therapies
12. Leber Congenital Amaurosis Emerging Drugs and Latest Therapeutic Advances
13. Leber Congenital Amaurosis Seven Major Market Analysis
14. Attribute Analysis
15. Leber Congenital Amaurosis Market Outlook (In US, EU5, and Japan)
16. Leber Congenital Amaurosis Companies Active in the Market
17. Leber Congenital Amaurosis Access and Reimbursement Overview
18. KOL Views on the Leber Congenital Amaurosis Market
19. Leber Congenital Amaurosis Market Drivers
20. Leber Congenital Amaurosis Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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