Leber Congenital Amaurosis (LCA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034

Leber Congenital Amaurosis (LCA) Market Outlook

Thelansis’s “Leber Congenital Amaurosis (LCA) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034" 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 (LCA) 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.

 

Geography coverage:

G8 (United States, EU5 [France, Germany, Italy, Spain, U.K.], Japan, and China)

Insights driven by robust research, including:

  • In-depth interviews with leading KOLs and payers
  • Physician surveys
  • RWE analysis for claims and EHR datasets
  • Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

  • Detailed Report (PDF)
  • Market Forecast Model (MS Excel-based automated dashboard)
  • Epidemiology (MS Excel; interactive tool)
  • Executive Insights (PowerPoint presentation)
  • Others: regular updates, customizations, consultant support

*As per Thelansis’s policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

  • 10-year market forecast (2024–2034)
  • Bottom-up patient-based market forecasts validated through the top-down sales methodology
  • Covers clinically and commercially-relevant patient populations/ line of therapies
  • Annualized drug-level sales and patient share projections
  • Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
  • Detailed methodology/sources & assumptions
  • Graphical and tabular outputs
  • Users can customize the model based on requirements

Key business questions answered:

  • How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
  • How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
  • What is the 10-year market outlook for sales and patient share?
  • Which events will have the greatest impact on the market’s trajectory?
  • What insights do interviewed experts provide on current and emerging treatments?
  • Which pipeline products show the most promise, and what is their potential for launch and future positioning?
  • What are the key unmet needs and KOL expectations for target profiles?
  • What key regulatory and payer requirements must be met to secure drug approval and favorable market access?
  • and more…


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