Leber Congenital Amaurosis Market

Market Overview:

The 7 major Leber congenital amaurosis markets are expected to exhibit a CAGR of 3.56% during 2025-2035.

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 2025-2035". 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, EU4 (Germany, Spain, Italy, and France), 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, 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.

Recent Developments:

• In June 2024, GenKOre announced a strategic agreement with Revvity's Gene Delivery franchise (previously known as SIRION Biotech GmbH), which specializes in viral vector-based gene delivery for gene and cell therapy. This research cooperation intends to discover effective gene therapy therapies for ophthalmic illnesses, including Leber congenital amaurosis 10 (LCA10).
• In November 2023, Atsena Therapeutics announced that the U.S. FDA had granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). The RMAT designation was obtained based on positive 6-month efficacy data from the company's ongoing Phase I/II clinical trial of ATSN-101.
• In September 2023, Ocugen, Inc. reported a clinical study update for retinitis pigmentosa patients treated in the Phase 1/2 trial to examine the safety and efficacy of OCU400 for retinitis pigmentosa with NR2E3 and Rhodopsin (RHO) mutations, as well as Leber congenital amaurosis with CEP290 gene mutation(s).

Key Highlights:

• Leber congenital amaurosis is a rare congenital eye condition that affects approximately 2-3 of every 100,000 live births.
• It is the second most prevalent inherited retinal dystrophy, accounting for approximately 5% of all retinal dystrophies.
• LCA is the main cause of blindness in children, accounting for approximately 20% of blindness in school-aged children.
• Patients typically begin losing their sight in childhood and become entirely blind in their twenties or thirties.
• Each child whose parents have LCA has a 25% chance of acquiring the condition.

Drugs:

Luxturna (voretigene neparvovec) is a gene therapy medication that is used to treat adults and children with Leber congenital amaurosis. Luxturna is injected beneath the retina and delivers a functional copy of the RPE65 gene to retinal cells. This causes the cells to create the RPE65 protein, which aids the visual cycle and converts light into electrical impulses that the brain can comprehend.

QR-110 is a first-in-class experimental RNA-based oligonucleotide that targets the underlying cause of Leber's congenital amaurosis 10: the p.Cys998X mutation in the CEP290 gene. QR-110 is intended to be administered using intravitreal injections in the eye and has received orphan drug designation in the United States and the European Union, as well as fast track status from the FDA.

EDIT-101 is a CRISPR/Cas9-based experimental medication being studied for the treatment of LCA10, which involves eliminating the IVS26 CEP290 mutant allele. EDIT-101 is given as a subretinal injection to reach and deliver gene editing equipment to photoreceptor cells.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

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 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the Leber congenital amaurosis market across the seven major markets in 2024 and what will it look like in 2035?
• 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 (​2019-2035​) of Leber congenital amaurosis across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Leber congenital amaurosis by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Leber congenital amaurosis by gender across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Leber congenital amaurosis by type across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with Leber congenital amaurosis across the seven major markets?
• What is the size of the Leber congenital amaurosis patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) 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?