Leber Congenital Amaurosis Market, By Treatment Type (Gene Therapy, Pharmaceutical Drugs, Retinal Prosthesis, Assistive Devices, and Others), By Target Gene (RPE65, GUCY2D, AIPL1, RPGRIP1, CEP290, and Other Genes), By End User (Hospitals, Specialized Eye Clinics, Ophthalmology Research Centers, Home Care Settings, and Others), By Geography (North America, Latin America, Europe, Asia Pacific, Middle East & Africa)

Published In : Aug 2024

Code : CMI7271

Pages :140

Formats :

Excel and PDF

Industry : Pharmaceutical

Market Concentration and Competitive Landscape

Leber Congenital Amaurosis Market Concentration By Players

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Growing Orphan Drug Designation

Orphan drug status provides valuable incentives for pharmaceutical companies to invest more resources into research and development of therapies for rare diseases. Obtaining such designation opens up several benefits like clinical trial cost reimbursement, tax credits, exclusive rights over the designated drug for seven years post approval among others. More drug makers are thus actively pursuing orphan designation for their pipeline drugs focused on niche rare disease markets like LCA which impacts only a miniscule population.

Over the past decade, the regulatory landscape around orphan drugs has also evolved significantly. Both FDA and EMA have further strengthened the incentives and streamlined their approval pathways for orphan medicines to encourage innovation in areas of high unmet need. Pharma firms can now attain orphan drug designation even during early clinical trial stages based on plausible hypothesis of drug's clinical benefit. Additionally, regulators recognise the practical challenges of conducting traditional large scale efficacy studies for rare diseases and are more open to accept surrogate markers and real-world evidence instead.

A visible outcome of these policy developments is the rising number of orphan drug designations being awarded year-on-year. Many major players in ophthalmology too have obtained the coveted status. This demonstrates the commercial interest that rare retinal dystrophies like LCA represent nowadays. It becomes lucrative for drugmakers to invest in research and win designation so that they can enjoy market exclusivity should their candidate therapy succeeds. Consequently, the LCA pipeline is growing rapidly with multiple drug candidates at various stages of clinical evaluation having already secured orphan status from regulatory agencies. Their expected approvals in the coming years will significantly expand treatment options for LCA patients. Therefore, the proliferating orphan drug activity puts the global LCA market on a firm footing for steady growth.

Key Players Insights