Autosomal Dominant Optic Atrophy Market
Key Highlights
- Autosomal Dominant Optic Atrophy (ADOA) market is expected to experience steady growth during the forecast period (2024–2034). This growth is a direct consequence of various market-driving factors such as growing awareness, advancements in diagnosis, and the launch of new therapeutic options.
- Currently, there is no approved therapy for ADOA. However, cholinesterase inhibitors, memantine, and cognitive stimulation therapy are widely used. Moreover, research is ongoing to explore potential therapies and other novel approaches.
- The treatment and diagnosis of ADOA include several unmet needs, including the absence of effective approved treatments to prevent vision loss, delays in diagnosis, an incomplete understanding of the disease's mechanisms, and limited data on the natural history and genotype-phenotype correlations. However, the ADOA market is expected to grow as key players in the emerging pipeline focus on developing tailored therapies and innovative treatment approaches, with both approved and emerging therapies driving market expansion during the forecast period (2024–2034).
- PYC Therapeutics and Stoke Therapeutics are developing promising drugs for the treatment of ADOA including PYC001 and STK-002, respectively. The introduction of these therapies is expected to drive significant market growth during the forecast period (2024-2034).
DelveInsight’s comprehensive report titled “Autosomal Dominant Optic Atrophy (ADOA) — Market Insights, Epidemiology, and Market Forecast – 2034” offers a detailed analysis of ADOA. The report presents historical and projected epidemiological data covering total diagnosed prevalent cases of ADOA, gene-specific diagnosed prevalent cases of ADOA, and treated cases of ADOA In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from study period (2020 to 2034).
The report analyzes the existing treatment practices and unmet medical requirements for ADOA. It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.
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Study Period |
2020 to 2034 |
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Forecast Period |
2024-2034 |
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Geographies Covered |
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ADOA Market |
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Market Analysis |
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ADOA Market players |
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ADOA Epidemiology |
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ADOA Overview
ADOA is a genetic disorder primarily caused by mutations in the OPA1 gene, which lead to mitochondrial dysfunction and subsequent degeneration of retinal ganglion cells. This results in progressive loss of vision in both eyes, typically beginning in early childhood, often between the ages of 4 and 6.
Common symptoms include defects in the central visual field, reduced color vision, and varying levels of visual impairment, which can potentially lead to blindness. The disease's pathophysiology involves mitochondrial dysfunction, causing increased oxidative stress and apoptosis of retinal cells.
ADOA Diagnosis and Treatment Algorithm
Diagnosis of ADOA typically involves a comprehensive assessment by an ophthalmologist, including family history, clinical examination, and genetic testing for OPA1 mutations.
Currently, there are no approved treatments to halt or reverse ADOA. The primary purpose of treatment is to manage symptoms and improve quality of life, as patients often rely on low-vision aids and supportive services due to the progressive nature of the disease.
Regular monitoring with annual assessments of visual acuity, visual fields, color vision, extraocular muscles, and hearing is recommended after diagnosis. Topical agents like brimonidine that may have neuroprotective or antiapoptotic effects on retinal ganglion cells have also been used, although evidence of their efficacy remains limited.
ADOA Epidemiology
The epidemiology section of the ADOA market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.
This section also presents the data with relevant tables and graphs, offering a clear and concise view of the prevalence of ADOA. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.
Key Findings
According to various literature analyses, the prevalence of ADOA in the US is estimated to be between 1 in 12 thousand to 1 in 50 thousand people.
As per analysis in the UK, in 2020 the prevalence of ADOA in the UK is approximately 1 in 35 thousand.
Based on our analysis, approximately 65-90% of dominant optic atrophy cases are caused by mutations in the OPA1 gene. Less common genetic subtypes include OPA4 and OPA5, which also cause autosomal dominant optic atrophy.
ADOA Market Outlook
ADOA is a genetically inherited condition characterized by the progressive loss of vision due to mitochondrial dysfunction in the optic nerve fibers. Currently, there is no cure for ADOA, but several treatments and research efforts are underway to address this condition.
Pharmacological treatments include idebenone, a supplement that has shown some improvement in visual acuity in patients with ADOA. Idebenone works by increasing the production of ATP, which helps to mitigate the effects of mitochondrial dysfunction. Off-label drugs, such as Coenzyme Q10, have also been used in some cases to support mitochondrial function and potentially slow the progression of the disease.
Non-pharmacological treatments focus on managing the symptoms and improving the quality of life for patients with ADOA. Low-vision aids such as magnifiers, telescopes, and tinted lenses can help individuals with ADOA to function better. Cochlear implants have been shown to improve audition in patients with sensorineural deafness, which is a common complication in some cases of ADOA.
Additionally, there are ongoing research efforts to develop novel treatments, such as STK-002, an antisense oligonucleotide (ASO) that targets the OPA1 gene, and PYC-001 that increases expression of the remaining healthy copy of OPA1 to compensate for the protein insufficiency. STK-002 has been shown to increase OPA1 protein levels in retinal ganglion cells (RGCs) after intravitreal administration in non-human primates, representing a potential disease-modifying therapy for ADOA patients.
With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, the ADOA market in the 7MM is expected to change significantly during the study period (2020–2034).
ADOA Drug Chapters
Emerging ADOA Drugs
The pipeline of ADOA is quite limited with few products available in the developmental stage. Key players involved in the development of promising products include PYC Therapeutics, Stoke Therapeutics, among others.
PYC001: PYC Therapeutics
PYC-001 is developed by PYC Therapeutics for the treatment of ADOA. It increases the expression of the OPA1 protein, crucial for retinal health, by targeting the OPA1 gene. Administered via intravitreal injection, PYC-001 aims to restore normal retinal function. Currently, the drug candidate is being investigated in a Phase I clinical trial, PYC-001 has shown a suitable safety profile and demonstrated disease-modifying potential in preclinical studies.
STK-002: Stoke Therapeutics
STK-002 is a proprietary antisense oligonucleotide (ASO) developed by Stoke Therapeutics for the treatment of ADOA. This rare disease causes progressive and irreversible vision loss in both eyes starting in the first decade of life. STK-002 aims to restore OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene, potentially slowing or stopping vision loss. The company plans to launch a Phase I clinical trial (OSPREY), for STK-002 in 2024, according to their recent presentation.
Note: Detailed emerging therapies assessment will be provided in the final report.
ADOA Market Segmentation
DelveInsight’s ‘ADOA Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a detailed outlook of the current and future ADOA market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.
ADOA Market Size by Countries
The ADOA market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2023, the United States held a significant share of the overall 7MM (Seven Major Markets) ADOA market, primarily attributed to the country's higher prevalence of the condition and the elevated cost of the available treatments. This dominance is projected to persist, especially with the potential early introduction of new products.
ADOA Market Size by Therapies
ADOA Market Size by Therapies is categorized into current and emerging markets for the study period (2020–2034). Emerging drugs anticipated to enter the ADOA market include PYC001 and STK-002, among other drug candidates.
Note: Detailed market segment assessment will be provided in the final report.
ADOA Drugs Uptake
This section focuses on the sales uptake of potential ADOA drugs that have recently been launched or are anticipated to be launched in the ADOA market between 2020 and 2034. It estimates the market penetration of ADOA drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the ADOA market.
The emerging ADOA therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the ADOA market.
Note: Detailed assessment of drug uptake and attribute analysis will be provided in the full report
ADOA Market Access and Reimbursement
DelveInsight’s ‘ADOA – Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a descriptive overview of the market access and reimbursement scenario of ADOA.
This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.
KOL Views
To keep up with current ADOA market trends and fill gaps in secondary findings, we interview KOLs and SMEs working in the ADOA domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or ADOA market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the ADOA's unmet needs.
ADOA: KOL Insights
DelveInsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as the University of California, Davis, University Hospital, Angers, and Sanford Health among others.
“ADOA, a rare genetic disorder, is a significant challenge in the US healthcare system. Treatment options are limited, and patients often face difficulties accessing appropriate care and support”.
“Early detection is crucial for timely intervention and management. However, limited awareness among healthcare professionals and lack of access to specialized genetic testing can delay diagnosis, leading to suboptimal patient outcomes”.
Note: Detailed assessment of KOL Views will be provided in the full report on the ADOA.
Competitive Intelligence Analysis
We conduct a Competitive and Market Intelligence analysis of the ADOA market, utilizing various competitive intelligence tools such as SWOT analysis and market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.
ADOA Pipeline Development Activities
The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for ADOA. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, patent details, and other information for emerging ADOA therapies.
ADOA Report Insights
- ADOA Patient Population
- Therapeutic Approaches
- ADOA Pipeline Analysis
- ADOA Market Size and Trends
- ADOA Market Opportunities
- Impact of Upcoming Therapies
ADOA Report Key Strengths
- 11 Years Forecast
- The 7MM Coverage
- ADOA Epidemiology Segmentation
- Key Cross Competition
- Highly Analyzed ADOA Market
- ADOA Drugs Uptake
ADOA Report Assessment
- ADOA Current Treatment Practices
- Unmet Needs
- ADOA Pipeline Product Profiles
- ADOA Market Attractiveness
Key Questions
- How common is an ADOA?
- What are the key findings of ADOA epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020–2034)?
- What are the currently available treatments for ADOA?
- What are the disease risks, burdens, and unmet needs of ADOA?
- At what CAGR is the ADOA market and its epidemiology expected to grow in the 7MM during the forecast period (2024–2034)?
- How would the unmet needs impact the ADOA market dynamics and subsequently influence the analysis of the related trends?
- What would be the forecasted patient pool of ADOA in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
- Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2024–2034)?
- How many companies are currently developing therapies for the treatment of ADOA?
Reasons to Buy
- The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the ADOA market.
- Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
- Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
- Distribution of historical and current patient share based on real-world prescription data in the US, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan.
- Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
- Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
- Highlights of access and reimbursement policies for ADOA, barriers to accessibility of approved therapy, and patient assistance programs.
- To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
- Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
