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. Leber Congenital Amaurosis 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 Leber Congenital Amaurosis genes (one from each parent) needed to cause the disorder.
Leber Congenital Amaurosis causes an abnormally low electrical response of the retina. An electrodiagnostic test known as an electroretinogram (ERG) is recommended to investigate how the retina works. The retina’s electrical activity is measured under different lighting conditions to determine which part of the retina is not functioning normally. This test requires the eyes to be dilated with special eye drops. A hard contact lens in each eye is also used to measure the eye’s responses to different kinds of light.
Multiple genetic mutations have been identified to cause Leber Congenital Amaurosis. Therefore, new gene therapies for Leber Congenital Amaurosis are under development. Gene therapy is currently available for Leber Congenital Amaurosis caused by two mutations in a gene called RPE65, which accounts for about 6% of Leber Congenital Amaurosis cases.
Leber Congenital Amaurosis Market Key Facts
According to the National Organization for Rare Disorders (NORD, 2021), the prevalence of Leber congenital amaurosis (LCA) has been estimated to be 1–2 per 100,000 births.
According to Coussa et al. (2017), Leber Congenital Amaurosis 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, Leber Congenital Amaurosis was reported to account for about 20% of legal blindness in children.
As per Feldhaus et al. (2020), the most frequent mutation in Leber Congenital Amaurosis patients from the large German cohort was c.2991+1655A>G – found in 87% of patients (20/23).
Genetic and Rare Diseases Information Center (2021) estimated that the number of people with Leber Congenital Amaurosis in the US is between 3,000 and 30,000.
As per the American Association for Pediatric Ophthalmology & Strabismus (2022), Leber Congenital Amaurosis 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 Leber Congenital Amaurosis.
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 details 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 –
Total Prevalent cases of Leber Congenital Amaurosis in the 7MM [2019–2032]
Total Diagnosed Prevalent cases of Leber Congenital Amaurosis in the 7MM [2019–2032]
Gene-specific cases of Leber Congenital Amaurosis in the 7MM [2019–2032]
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
There is no approved treatment for other types of Leber Congenital Amaurosis at present, but several clinical trials are ongoing. In the meantime, treatment is focused on alleviating symptoms and optimizing remaining sight by treating the other eye conditions associated with Leber Congenital Amaurosis.
Some people with Leber Congenital Amaurosis may also benefit from low-vision aids, including electronic, computer-based, and optical aids. Low vision aids to maximize visual function can be very useful for patients with Leber Congenital Amaurosis. Some children with Leber Congenital Amaurosis suffer from conditions affecting other parts of the body (like Joubert syndrome or Senior-Loken syndrome). They need input from various specialists coordinated by the pediatrician.
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 Leber Congenital Amaurosis called LCA2. Luxturna is manufactured by Spark Therapeutics.
Several major pharma and biotech companies are actively involved in the development of therapies for Leber Congenital Amaurosis. The key players involved in developing new therapies to treat Leber Congenital Amaurosis include ProQR Therapeutics, Atsena Therapeutics, and others. Notably, ProQR Therapeutics stands out among them, with Leber Congenital Amaurosis drug candidates advancing to the most advanced stage, specifically phase II/III of clinical trials.
Leber Congenital Amaurosis Companies Actively Working in the Therapeutics Market Include
Applied Genetic Technologies
And Many Others
Emerging and Marketed Leber Congenital Amaurosis Therapies Covered in the Report Include:
Sepofarsen (QR-110): ProQR Therapeutics
EDIT-101: Editas Medicine, Inc.
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
22. DelveInsight Capabilities
*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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