Leber Congenital Amaurosis Market Size and Trends

The Global Leber Congenital Amaurosis Market is estimated to be valued at USD 1.23 Bn in 2024 and is expected to reach USD 1.68 Bn by 2031, exhibiting a compound annual growth rate (CAGR) of 4.5% from 2024 to 2031.

Leber Congenital Amaurosis Market Key Factors

To learn more about this report, request sample copy

Increasing prevalence of inherited retinal diseases

The prevalence of inherited retinal diseases is increasing at an alarming rate across the globe. Leber congenital amaurosis, being one form of inherited retinal dystrophy, is also witnessing a spike in cases year-over-year. As per some estimations, around 2-3 out of every 100,000 live births are diagnosed with LCA. Given that the global birth rate averages around 140 million newborns annually, it means around 2,800 to 4,200 new LCA cases are emerging each year.

This upward trend can majorly be attributed to the advancing age of prospective parents. Medical science has enabled people to delay parenthood till late 30s or early 40s now unlike earlier times when most people had kids in their 20s. However, it is a well-established fact that the likelihood of inherited genetic disorders and mutations increase with increasing parental age. Studies have shown advanced paternal age to be a significant risk factor for several inherited eye diseases including LCA. Since more people are opting to become parents at an older age nowadays due to social and economic reasons, the chances of their offspring inheriting genetic retinal conditions like LCA grow correspondingly.

Another important reason for the rise in inherited retinal dystrophy prevalence is improving diagnosis. Availability of advanced genetic testing tools and techniques allows for faster and more definitive diagnosis of even rare retinal disorders. Previously, conditions like LCA which manifest from childhood often went undiagnosed for years due to the lack of expertise and diagnostic limitations. But now sophisticated genetic screening methods enable diagnosis even in newborn babies showing first signs of visual impairment. This has led to more accurate disease surveillance worldwide. Also, growing awareness about these genetic conditions thanks to patient advocacy groups is encouraging more families to go for diagnostic screening of family members, leading to identification of new cases.

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.