ABCA4 Retinopathy Therapeutics Market
Number of pages: 100 | Report Format: PDF | Published date: May 03, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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CAGR |
18.6% |
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Base Year for Estimation |
2022 |
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Forecast Period |
2023 to 2031 |
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Historical Year |
2021 |
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Segments Covered |
Drug types, Age group, Distributional channels, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global ABCA4 retinopathy therapeutics market is expected to register a revenue CAGR of 18.6% from 2023 to 2031.
ABCA4 Retinopathy Therapeutics Market Fundamentals
ABCA4 retinopathy therapeutics is a genetic disorder that affects the retina. This inherited genetic disorder, also known as Stargardt disease, is caused by mutations in the ABCA4 gene. The disease affects most of the retinal region of the macula, which is responsible for the eye's clear and centered vision. ABCA4 Retinopathy is a juvenile macular degeneration and may cause progressive vision loss. The gene responsible for the photoreceptor-specific ATP-binding cassette (ABC) transporter (ABCA4) is altered in ABCA4 retinopathy, an autosomal recessive maculopathy with early and young-adult onset.
Unfortunately, there is currently no cure for ABCA4 retinopathy, but several treatment options can help slow the progression of the disease and manage its symptoms. However, encouraging research & development efforts in creating novel drugs for treating and managing patients suffering from ABCA4 retinopathy-related vision loss have been made. Several drugs are being investigated in clinical trials for their potential in treating this condition.
Millions of people worldwide have inherited retinal diseases. Globally, more than 2 million people are affected by inherited retinal diseases, the leading cause of vision loss in the working-age population. In the U.K., inherited retinal diseases are the primary cause of blindness in adults and a secondary cause in children.
ABCA4 Retinopathy Therapeutics Market Dynamics:
The global ABCA4 retinopathy therapeutics market revenue is driven by the rising number of ABCA4 retinopathy cases Furthermore, the ongoing research and development activities to develop novel drug therapies and advanced treatments are further boosting the global market ABCA4 retinopathy revenue. Moreover, increasing focus on health, financial support for medical research, positive results of clinical trials, and expanding awareness of inherited diseases are expected to act as key factors in ABCA4 retinopathy therapeutics market revenue share future.
Several companies are developing gene therapies that aim to deliver a functional copy of the ABCA4 gene to the affected cells in the retina. This approach has shown promise in early-stage clinical trials. Genetic research has made significant progress in recent years, leading to a better understanding of the underlying causes of ABCA4 retinopathy. This has enabled the development of more targeted therapies that can address the specific genetic mutations responsible for the disease. Another approach being explored is CRISPR/Cas9 gene editing, which involves using a molecular tool to edit the defective ABCA4 gene in affected cells. This approach has shown potential in preclinical studies.
Market players significantly contribute to creating a potent treatment option for ABCA4 retinopathy. For instance, on January 11, 2021, the fishing and biotech company Aker BioMarine signed a contract with biotech entrepreneur Dr. Michael Davidson to create pharmaceutical treatments for conditions affecting the brain and eyes, such as Alzheimer's disease, dry age-related macular degeneration, and Stargardt's juvenile blindness, using the LYSOVETA process, developed by Aker BioMarine.
However, the time intensive research, unavailability of effective treatment, unawareness about the disease among the population, and lack of skilled hospital staffs and other healthcare professionals, are expected to restrain market revenue growth. Clinical development of therapies for ABCA4 retinopathy is complex and challenging, due to the rarity of the disease and the complexity of the underlying genetic mutations. Therefore, companies must invest significant resources in clinical trials to demonstrate safety and efficacy and gain regulatory approval.
ABCA4 Retinopathy Therapeutics Market Ecosystem
The global ABCA4 retinopathy therapeutics market is analyzed from four perspectives: drug types, age group, distributional channels, and region.
ABCA4 Retinopathy Therapeutics Market Segmentation by Drug Type
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Based on drug type, the global ABCA4 retinopathy therapeutics market is segmented into anti-VEGF drugs, LBS-008, and others.
The LBS 008 drug segment, if completed the clinical trials and approved, is expected to be the fastest-growing segment in the global ABCA4 retinopathy market during the forecast period. This rise in revenue is expected as the drug LBS 008 (also known as Tinlarebant) is initiated for phase 3 clinical trial. LBS 008 is an oral medication that needs to be taken once daily to prevent the accumulation of vitamin A-based toxins. This results in a decrease in harmful vitamin A metabolites (bis retinoids), which have been linked to the beginning and development of ABCA4 retinopathy therapeutics.
Anti-VEGF drugs can be used as monotherapy for the treatment of ABCA4 retinopathy. This type of treatment involves the use of a single drug to inhibit the growth of abnormal blood vessels in the retina. Anti-VEGF monotherapy may be effective in slowing the progression of the disease and improving visual function in some patients.
Other drugs such as Emixustat (under clinical trial) alters the visual cycle by preventing retinal pigment epithelium protein 65 (RPE65), an essential enzyme in this pathway. In human clinical investigations, oral emixustat was discovered to be generally well tolerated, with delayed dark adaption being the most frequent side effect.
ABCA4 Retinopathy Therapeutics Market Segmentation by Age Group
Based on age group, the global ABCA4 retinopathy therapeutics market is segmented into children and adults.
The segment for children is dominating the global market with the largest revenue share. This large revenue share is because the ABCA4 retinopathy is usually noticed within the first or second decades of life, experiencing a severe vision loss, which in nearly all cases progresses to irreversible decreased visual acuity.
The adult segment is expected to show a significant revenue share during the forecast period as the rising number of adult patients are diagnosed with ABCA4 Retinopathy and experiencing vision loss.
ABCA4 Retinopathy Therapeutics Market Segmentation by Distribution Channel
Based on the distribution channel, the global ABCA4 retinopathy therapeutics market is segmented into hospital pharmacy, online pharmacy, and retail pharmacy.
Hospital pharmacy is expected to dominate with a significant revenue share during the forecast period. This large revenue share can be due to the large number of patients visiting the hospital for the required diagnosis and treatment and hence buying most of the medicines from the hospitals. Furthermore, these are available in the remote or low-income areas, providing medicines to patients residing in these regions as well.
The online pharmacy segment is expected to be the fastest growing segment in the global ABCA4 retinopathy market during the forecast period. The segment’s growth can be attributed to the wide accessibility, convenient option, and better price range. Additionally, online pharmacy collaborate closely with licensed local chemists and online doctors. In developed nations like the U.S., medicines by online pharmacies are distributed to the population with more reliability. Furthermore, there has been a growing adoption of online pharmacies in developing nations, which is expected to create an environment that is favorable for their expansion during the forecast period.
The retail pharmacy segment accounted for the second-largest revenue share during the forecast period. Opportunities for market growth are created by the increase in the distribution of ABCA4 retinopathy medications given through retail pharmacies and the increase in the number of retail pharmacies in advanced nations. Additionally, due to their accessibility, patients prefer to buy their medications from retail pharmacies.
ABCA4 Retinopathy Therapeutics Market Segmentation by Region
Based on region, the ABCA4 retinopathy therapeutics market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa.
North America, with the largest revenue share, is expected to dominate the global market during the forecast period. The high revenue share is attributed to the numerous laboratories and companies performing significant research and development activities, well-established research labs, high healthcare spending, strong regulatory framework, and a large patient population. The growing occurrence of ABCA4 retinopathy is expected to boost the market revenue growth during the forecast period. Additionally, the presence of qualified personnel, product manufacturers, significant investment in research and development, and substantial healthcare spending contribute to the regional revenue growth of the ABCA4 retinopathy therapeutics market.
Asia Pacific accounts for the second-largest revenue share in the global retinopathy market and is expected to rise significantly during the forecast period. This market revenue growth in the coming years is expected due to the increased R&D activities for an effective ABCA4 therapy, large population base, rising awareness about genetic diseases, developing infrastructure, robust business models, and competition among top companies.
Competitive Landscape of ABCA4 Retinopathy Therapeutics Market
A list of prominent players operating in the global ABCA4 retinopathy therapeutics market includes:
Numerous companies give priority to methods of organic development such as the continual optimization of commercial activities, reallocating funds into the creation of high-profit products that support growth and revenues, the creation of new business models, or the development of new products and services. Other methods of development include new product launches, product approvals, patents, and events. Three different categories of market-based strategic growth techniques include acquisitions, partnerships, and collaborations. Market players might expand their consumer bases and business operations as a result of these actions.
Strategic Developments in ABCA4 Retinopathy Therapeutics Market
ABCA4 retinopathy is an inherited genetic disorder caused by the mutation of the ABCA4 gene. The disease affects juvenile macular degradation and the retina, resulting in vision loss.
The expected revenue CAGR of the global ABCA4 Retinopathy therapeutics market is 18.6% during the forecast period 2023-2031.
The leading companies operating in the global ABCA4 retinopathy therapeutics market are Alkeus Pharmaceuticals, Sanofi SA, Stargazer Pharmaceuticals, Inc, Kubota Vision Inc., and Astellas Pharma Inc.
The rising number of ABCA4 retinopathy cases, collaborations, and acquisitions between market players, the ongoing research and development activities to develop novel drug therapies, and expanding awareness of inherited diseases among the population are the factors driving the global ABCA4 retinopathy therapeutics market revenue growth.
North America accounts for the largest market revenue share in the ABCA4 retinopathy therapeutics.
*Insights on financial performance are subject to the availability of information in the public domain