Leber Congenital Amaurosis (LCA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Leber Congenital Amaurosis (LCA) Market Outlook

Thelansis’s “Leber Congenital Amaurosis (LCA) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Leber Congenital Amaurosis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Leber Congenital Amaurosis (LCA) Overview

Leber congenital amaurosis (LCA) is a vision disorder that primarily impacts the retina, the specialized tissue at the rear of the eye responsible for light and color detection. LCA can arise from mutations in a minimum of 14 genes, all essential for normal vision. The primary culprits behind this disorder are mutations in the CEP290, CRB1, GUCY2D, and RPE65 genes, while other gene mutations account for a smaller portion of cases. In uncommon instances, LCA is inherited as an autosomal dominant genetic condition. Presently, mutations in three genes, CRX, IMPDH1, and OTX2, are linked to this type of LCA. LCA is marked by a significant decline in visual acuity (equal to or less than 20/400) or blindness within the first year of life. Depending on the genetic basis, symptoms may include sluggish pupillary responses, wandering eye movements, sensitivity to light, high hyperopia, nystagmus, convergent strabismus, or keratoconus. A distinctive sign known as Franceschetti's oculo-digital sign, characterized by eye poking, pressing, and rubbing, is a defining feature. LCA can also be associated with gene mutations related to syndromes featuring neurodevelopmental delay, intellectual impairment, oculomotor apraxia-like difficulties in eye movement, and kidney dysfunction. The FDA has approved Voretigene neparvovec-rzyl (LuxturnaTM) to treat Leber congenital amaurosis (LCA). This marks a groundbreaking milestone as it is the first gene therapy approved by the FDA to address a disease. The treatment involves injecting a new, functional gene copy under the retina to target the appropriate cells.

• Leber congenital amaurosis affects 2 to 3 newborns out of every 100,000. It's one of the most common causes of childhood blindness.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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