Prachi
The global Leber Congenital Amaurosis (LCA) market is expected to witness high growth due to the recent advancements in gene therapy. LCA is an inherited retinal dystrophy that causes severe visual impairment in children. The market consists of several gene therapy products that target the underlying genetic causes of LCA.
The LCA market is primarily driven by the growing prevalence of genetic retinal disorders and vision loss in children. LCA affects around 2-3 people per 100,000 worldwide. The symptoms include severe vision loss at birth or within the first few months of life, followed by nystagmus and potentially cortical visual impairment. Currently, there is no approved treatment for all genetic subtypes of LCA. However, gene therapies are transforming the treatment landscape by targeting the root cause of the disease.
The global LCA market is estimated to be valued at US$ 1.23 billion in 2024 and is expected to exhibit a CAGR of 4.5% over the forecast period 2024 to 2031.
Key Takeaways
Key players operating in the LCA market are Spark Therapeutics, MeiraGTx Holdings plc, ProQR Therapeutics N.V., AGTC.
Leber Congenital Amaurosis Market Size is growing prevalence of inherited retinal diseases and orphan status of therapies are fueling the demand. Several gene therapy candidates are in clinical trials which is expected to receive approvals in the coming years.
Novel gene therapy approaches like CRISPR are enabling editings of multiple genes simultaneously. Companies are developing vision restoration therapies through optogenetics which stimulate retinal cells with light.
Market Trends
Gene therapy is transforming the treatment landscape with personalized treatments tailored for each genetic subtype of LCA. Several late stage gene therapy pipelines are expected to launch commercially.
Regulatory bodies are fast-tracking therapies for rare retinal conditions to improve patient access. The orphan drug designation provides market exclusivity to winning candidates.
Market Opportunities
Asia Pacific region with large patient population and growing healthcare infrastructure presents high growth opportunities. Partnering with regional biotech firms allow global players to penetrate emerging Asian markets.
Developing economies offer cost-effective clinical trials and manufacturing facilities attracting investments in retinal gene therapy research.
Impact of COVID-19 on Leber Congenital Amaurosis Market Growth
The outbreak of COVID-19 pandemic has significantly impacted the growth of Leber Congenital Amaurosis market. During the initial phase of lockdowns imposed across various countries, there was disruption in supply chain and logistics which hampered the availability of therapeutic drugs and devices. This led to delay in treatments and diagnosis of new patients suffering from Leber Congenital Amaurosis. Many ophthalmology clinics and hospitals also postponed routine check-ups and non-emergency eye procedures to focus on COVID-19 patients burdening the healthcare systems.
With social distancing norms and fear of virus transmission, patients started avoiding visits to hospitals and clinics for routine eye exams. This acted as a major roadblock for screening of new cases of Leber Congenital Amaurosis. Furthermore, nationwide lockdowns enforced social restrictions forcing people to stay indoors. This provided less scope of light exposure critical for vision development in infants suffering from Leber Congenital Amaurosis. All these factors collectively slowed down the market growth during initial months of pandemic.
However, with relaxation of lockdowns and adaptation of new safety protocols, ophthalmology centres have resumed operations. Greater availability of therapeutic drugs coupled with innovations in drug delivery have ensured continuity of treatments. Rising awareness among people about adhering to social distancing and hygiene measures has instilled confidence to seek regular medical care without fear of infections. Tele-ophthalmology is finding increased adoption to remotely examine and diagnose patients. Thus, market is recuperating steadily and expected to rebound in coming years driven by approval of novel gene therapies and sustained demand for current standard of care drugs…
North America accounts for the largest share of Leber Congenital Amaurosis market in terms of value. This is attributed to higher prevalence of the condition, growing gene therapy landscape and strong healthcare infrastructure in countries like US and Canada. Availability of advanced diagnostic and therapeutic options paired with favourable reimbursement framework further supplements the market growth. Europe is another lucrative market owing to rising R&D investments and increasing acceptance of novel therapies. Meanwhile, Asia Pacific region is poised to emerge as the fastest growing regional market for Leber Congenital Amaurosis over the forecast period. This is credited to escalating cases along with improving healthcare expenditures and health awareness in developing nations like China and India.
Germany holds the leading position in European Leber Congenital Amaurosis market led by presence of major pharmaceutical companies involved in drug development initiatives. Growing research collaborations and clinical trials are positive drivers. Besides,UK and France also contribute significantly to regional market share on back of expanding gene therapy trials, robust drug pipelines and supportive national health programs. China and Japan dominate Asia Pacific region due to large patient population base, improving clinical trial landscape and rise of local ophthalmic drug makers.
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