X-Linked Retinitis Pigmentosa Market

• The total retinitis pigmentosa market size in the US was estimated to be nearly USD 270 million in 2023, which is expected to increase due to the launch of novel emerging therapies.
• As per DelveInsight’s estimate, the total prevalent cases of retinitis pigmentosa in the 7MM were ~274,100 in 2023. These cases are expected to grow during the forecast period.
• The sub-types of non-syndromic retinitis pigmentosa include autosomal dominant retinitis pigmentosa, autosomal recessive retinitis pigmentosa, X-Linked Retinitis Pigmentosa, sporadic/simplex, Leber congenital amaurosis (LCA). 
• Non-syndromic retinitis pigmentosa accounts for ~65% of retinitis pigmentosa. Further, autosomal recessive accounts for ~10–15%, autosomal dominant accounts for 20–25%, and X-Linked Retinitis Pigmentosa is the rarest form of non-syndromic retinitis pigmentosa with early-onset and severe vision loss.
• According to DelveInsight, in the 7MM, X-Linked Retinitis Pigmentosa accounted for ~15% of the non-syndromic retinitis pigmentosa with ~16,500 cases in 2023.
• X-Linked Retinitis Pigmentosa, primarily caused by mutations in the RPGR gene, is a severe condition with a high incidence. Due to its impact, finding an effective treatment has garnered significant interest from the scientific community in recent years. Although several ocular gene therapy trials for X-Linked Retinitis Pigmentosa have been conducted or are in progress, there is currently no approved treatment. 
• Emerging players such as MeiraGTx/ Johnson & Johnson Innovative Medicine, Beacon Therapeutics, 4D Molecular Therapeutics, and others are evaluating their X-Linked Retinitis Pigmentosa therapies. Frontera Therapeutic’s FT-002 gene therapy for RPGR gene mutation-associated X-Linked Retinitis Pigmentosa is in the early clinical phase. 
• In June 2024, Beacon Therapeutics announced the treatment of the first patient in its registrational VISTA clinical trial with its lead asset, AGTC-501. Beacon Therapeutics plans to use the data generated from the VISTA trial, in combination with data from the Phase I/II HORIZON and Phase II SKYLINE studies, including all available long-term data, to support a BLA in the US and an MAA in Europe for the treatment of patients with XLRP.
• In its Q2 2024 financial results and operational highlights, 4D Molecular Therapeutics reported that program updates of 4D-125 for XLRP are expected in Q4 2024.
• In February 2024, Beacon Therapeutics announced the presentation of 12-month interim safety and efficacy results for the Phase II SKYLINE trial in patients with XLRP at the 47th Annual Macula Society Meeting. AGTC-501 was well tolerated and there were no clinically significant safety events associated with treatment, and any treatment-related adverse events were mostly nonserious and mild to moderate in severity.
• In March, MeiraGTx received a USD 50 million milestone after initiation of the extension study for the Phase III LUMEOS clinical trial for bota-vec for the treatment of XLRP.

DelveInsight's “X-Linked Retinitis Pigmentosa Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of historical and forecasted epidemiology as well as the X-Linked Retinitis Pigmentosa therapeutics market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

X-Linked Retinitis Pigmentosa market report provides current treatment, emerging therapies, market share of individual therapies, and historical and forecasted 7MM X-Linked Retinitis Pigmentosa market size from 2020 to 2034. The report also covers current X-Linked Retinitis Pigmentosa treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s potential.

X-Linked Retinitis Pigmentosa Treatment Market

X-Linked Retinitis Pigmentosa Overview

X-linked retinitis pigmentosa (X-Linked Retinitis Pigmentosa) is an incurable genetic disease that causes blindness. The disease is caused by a defect in the RPGR gene located on the X chromosome, which is why the disease affects men and women differently. Sight loss in X-Linked Retinitis Pigmentosa begins with ‘night blindness’ (i.e., loss of night vision) in adolescence, followed by a gradual loss of peripheral vision which results in progressively worsening ‘tunnel vision’. Ultimately, central vision is lost by the fourth decade, with most X-Linked Retinitis Pigmentosa patients becoming legally blind by the age of 40. 

X-Linked Retinitis Pigmentosa Diagnosis

The diagnostic journey in families with a retinitis pigmentosa history typically commences with thorough genetic counseling, assessing risks, and testing options. Prenatal genetic tests, such as CVS or amniocentesis, may be considered for fetal genetic analysis. After birth, a crucial symptom assessment by an ophthalmologist takes place. While initial retinitis pigmentosa symptoms commonly emerge in young adulthood, delays in referral and diagnosis often postpone confirmation until the age of 35.1 years. Genetic testing then identifies specific mutations. A comprehensive ophthalmologic evaluation, including fundoscopic examination, is indispensable, with ERG and visual field tests significantly contributing to understanding and guiding management.

Further details related to country-based variations in diagnosis are provided in the report…

X-Linked Retinitis Pigmentosa Treatment

Treatment of X-Linked Retinitis Pigmentosa remains challenging, and current treatments are not effective enough in restoring vision. Gene therapy of X-Linked Retinitis Pigmentosa, capable of restoring the functional RPGR gene, showed promising results in preclinical studies and clinical trials; however, to date, no approved product has entered the market. The most widely recommended treatment for many years to treat retinitis pigmentosa has been supplementation with vitamin A, which some studies have shown to slow the rate of retinal deterioration. 

X-Linked Retinitis Pigmentosa Epidemiology

The X-Linked Retinitis Pigmentosa epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of retinitis pigmentosa, total prevalent cases of X-Linked Retinitis Pigmentosa, gender-specific cases of X-Linked Retinitis Pigmentosa, age-specific cases of X-Linked Retinitis Pigmentosa, and prevalence of X-Linked Retinitis Pigmentosa by mutant genes in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• In the US, the total number of prevalent cases of retinitis pigmentosa was ~113,200 in 2023. Whereas, X-Linked Retinitis Pigmentosa in the US accounted for ~11,000 cases in 2023.
• Non-syndromic retinitis pigmentosa is more prevalent than syndromic retinitis pigmentosa, accounting for ~65% of retinitis pigmentosa cases. 
• Men are affected slightly more often than women due to the X-linked form being expressed more frequently in males.
• Among the EU4 countries, the highest number of prevalent cases of X-Linked Retinitis Pigmentosa in 2023 was found in Germany. While Spain accounted for the lowest number of prevalent cases. 

Stay Informed on X-Linked Retinitis Pigmentosa Epidemiology! Access comprehensive data on demographics and disease burden for effective healthcare planning.

X-Linked Retinitis Pigmentosa Drug Chapters

The drug chapter segment of the X-Linked Retinitis Pigmentosa report encloses a detailed analysis of X-Linked Retinitis Pigmentosa early and mid-stage (Phase I/II, and Phase II/III) pipeline drugs. It also deep dives into the X-Linked Retinitis Pigmentosa pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations. 

X-Linked Retinitis Pigmentosa Emerging Drugs

AGTC-501: Beacon Therapeutics

AGTC-501 is a gene therapy program currently in a Phase II/III clinical trial for the treatment of X-Linked Retinitis Pigmentosa that was acquired as part of Syncona’s acquisition of AGTC in November 2022. X-Linked Retinitis Pigmentosa is predominantly caused by mutations in the RPGR gene. Unlike other approaches in the space, AGTC-501 expresses the full-length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by X-Linked Retinitis Pigmentosa, including both rod and cone loss.

AGTC-501 has been awarded an Innovative Medicine Designation (ILAP) in the UK, Priority Medicine (PRIME) in the EU, and Fast Track in the US for the treatment of X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene.

4D-125: 4D Molecular Therapeutics

4D-125 is an investigational genetic medicine using the R100 vector for the treatment of X-Linked Retinitis Pigmentosa related to mutations in the RPGR gene. This product candidate is highly differentiated because it utilizes the primate-evolved and customized intravitreal R100 vector for transgene (RPGR) delivery to the retina with the goal of transgene expression in all regions of the retina after a single dose. 4D-125 is currently being evaluated in a Phase I/II trial for the treatment of X-Linked Retinitis Pigmentosa.

In January 2022, 4D Molecular Therapeutics announced that the US FDA had granted Fast Track Designation for 4D-125 for the treatment of patients with IRDs due to defects in the RPGR gene, including X-Linked Retinitis Pigmentosa.

Note: Detailed emerging therapies assessment will be provided in the final report...

X-Linked Retinitis Pigmentosa Drug Class Insights

There are currently no effective treatments available for X-Linked Retinitis Pigmentosa, but companies are developing a new technique of gene therapy which they believe may help to slow or even stop the degeneration. Several ocular gene therapy trials for X-Linked Retinitis Pigmentosa have been conducted or are in progress. Gene therapy is a powerful therapeutic strategy for recessive single-gene retinal degeneration. The pipeline currently includes drug classes such as AAV vector gene therapy (Botaretigene sparoparvovec, AGTC-501), and R100 vector gene therapy (4D-125). 

X-Linked Retinitis Pigmentosa Market Outlook

X-Linked Retinitis Pigmentosa is a severe form of retinal dysfunction without an approved effective treatment to date. Available therapies for retinitis pigmentosa, at their best, have just been able to delay retinal degeneration by reducing retinal cell death, improving retinal cell function, replacing cells, or creating an artificial retina. X-Linked Retinitis Pigmentosa, which is reported with considerable prevalence and severe manifestations, has been studied for gene therapy treatment; fortunately, promising developments in this area have been reported in recent years. Gene therapy using adeno-associated viral vectors is currently the most promising therapeutic approach.

Key players under development include Beacon Therapeutics (AGTC-501), 4D Molecular Therapeutics (4D-125), MeiraGTx/ Johnson & Johnson Innovative Medicine (Botaretigene sparoparvovec), and others. 

• The total market size in the US for retinitis pigmentosa was estimated to be nearly USD 270 million in 2023, which is expected to increase due to the launch of novel emerging therapies.
• Among the therapies being developed for X-Linked Retinitis Pigmentosa, Botaretigene sparoparvovec is expected to capture the largest market size in 2034. 

Key Updates

• In June 2024, Beacon Therapeutics announced the treatment of the first patient in its registrational VISTA clinical trial with its lead asset, AGTC-501. Beacon Therapeutics plans to use the data generated from the VISTA trial, in combination with data from the Phase I/II HORIZON and Phase II SKYLINE studies, including all available long-term data, to support a BLA in the US and an MAA in Europe for the treatment of patients with X-Linked Retinitis Pigmentosa.
• In its Q2 2024 financial results and operational highlights, 4D Molecular Therapeutics reported that program updates of 4D-125 for X-Linked Retinitis Pigmentosa are expected in Q4 2024. 
• In February 2024, Beacon Therapeutics announced the presentation of 12-month interim safety and efficacy results for the Phase II SKYLINE trial in patients with X-Linked Retinitis Pigmentosa at the 47^th Annual Macula Society Meeting. AGTC-501 was well tolerated and there were no clinically significant safety events associated with treatment, and any treatment-related adverse events were mostly nonserious and mild to moderate in severity.
• In March, MeiraGTx received a USD 50 million milestone after initiation of the extension study for the Phase III LUMEOS clinical trial for bota-vec for the treatment of X-Linked Retinitis Pigmentosa.

X-Linked Retinitis Pigmentosa Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024–2034, which depends on the competitive landscape, safety, efficacy data, and order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake. 

X-Linked Retinitis Pigmentosa Activities

The report provides insights into X-Linked Retinitis Pigmentosa clinicsl trials within Phase III, Phase II/III, and Phase I/II. It also analyzes key players involved in developing targeted therapeutics. 

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for X-Linked Retinitis Pigmentosa emerging therapies. 

Explore the latest insights into the X-Linked Retinitis Pigmentosa pipeline, emerging therapies, and future treatment potential. Stay informed today!

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including Medical/scientific writers, Ophthalmologists, Professors, and others.

DelveInsight’s analysts connected with 40+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the Foundation Fighting Blindness, the American Academy of Ophthalmology, the American Optometric Association, the School of the Johns Hopkins University Wilmer Eye Institute, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or X-Linked Retinitis Pigmentosa market trends. 

Qualitative Analysis

We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and conjoint analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. In conjoint analysis, the analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health technology assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug. In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs including Medicare, Medicaid, Health Insurance Program (CHIP), and the state and federal health insurance marketplaces are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs), and third-party organizations that provide services, and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the X-Linked Retinitis Pigmentosa Market Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of X-Linked Retinitis Pigmentosa, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines.
• Additionally, an all-inclusive account of the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will impact the current treatment landscape.
• A detailed review of the XLPR market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM X-Linked Retinitis Pigmentosa Market.

X-Linked Retinitis Pigmentosa Report Insights

• X-Linked Retinitis Pigmentosa Patient Population
• X-Linked Retinitis Pigmentosa Therapeutic Approaches
• X-Linked Retinitis Pigmentosa Pipeline Analysis
• X-Linked Retinitis Pigmentosa Market Size and Trends
• Existing and future Market Opportunity 

X-Linked Retinitis Pigmentosa Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage 
• X-Linked Retinitis Pigmentosa Epidemiology Segmentation
• Key Cross Competition 
• Conjoint analysis
• X-Linked Retinitis Pigmentosa Drugs Uptake
• Key X-Linked Retinitis Pigmentosa Market Forecast Assumptions

X-Linked Retinitis Pigmentosa Report Assessment

• Current X-Linked Retinitis Pigmentosa Treatment Practices
• X-Linked Retinitis Pigmentosa Unmet Needs
• X-Linked Retinitis Pigmentosa Pipeline Product Profiles
• X-Linked Retinitis Pigmentosa Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)
• X-Linked Retinitis Pigmentosa Market Drivers
• X-Linked Retinitis Pigmentosa Market Barriers

FAQs

• What is the historical and forecasted X-Linked Retinitis Pigmentosa patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?
• What was the X-Linked Retinitis Pigmentosa total market size, the market size by therapies, market share (%) distribution in 2023, and what would it look like in 2034? What are the contributing factors for this growth?
• What are the pricing variations among different geographies for off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

• Although multiple expert guidelines recommend testing for targetable mutations before therapy initiation, why do barriers to testing remain high?
• What are the current and emerging options for treating X-Linked Retinitis Pigmentosa? 

• How many companies are developing therapies to treat X-Linked Retinitis Pigmentosa?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies? 
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?

Reasons to buy X-Linked Retinitis Pigmentosa Market Forecast Report

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the X-Linked Retinitis Pigmentosa 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 along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Highlights of access and reimbursement policies, barriers to accessibility of expensive off-label therapies, 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.