(Albany, USA) DelveInsight’s “Retinitis Pigmentosa Market Insights, Epidemiology, and Market Forecast 2034” report delivers an in-depth understanding of historical and forecasted epidemiology as well as the Retinitis Pigmentosa market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
The Retinitis Pigmentosa market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Retinitis Pigmentosa market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Retinitis Pigmentosa treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Retinitis Pigmentosa market.
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Key Takeaways from the Retinitis Pigmentosa Market Report
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Retinitis Pigmentosa Overview
Retinitis Pigmentosa is a group of rare genetic disorders that cause progressive retinal degeneration and vision loss. Retinitis Pigmentosa symptoms typically begin with night blindness, followed by a gradual loss of peripheral vision, leading to tunnel vision. Retinitis Pigmentosa causes are usually linked to inherited genetic mutations that affect the retina’s ability to respond to light. Retinitis Pigmentosa diagnosis is often made through a combination of genetic testing, electroretinography (ERG), visual field testing, and optical coherence tomography (OCT).
Retinitis Pigmentosa treatment currently focuses on managing symptoms and slowing progression, with options including vitamin A supplements, retinal implants, and emerging gene therapies. Retinitis Pigmentosa clinical trials are actively exploring innovative therapies, such as stem cell treatments and CRISPR gene editing, to restore or preserve vision. Retinitis Pigmentosa prognosis varies based on the genetic subtype, with some patients maintaining functional vision into adulthood, while others experience severe impairment earlier.
Retinitis Pigmentosa awareness is essential for early detection and support. Retinitis Pigmentosa support groups, along with genetic counseling, help patients and families navigate the challenges of this lifelong condition. With advancing research, the future of Retinitis Pigmentosa management continues to show promise for improved outcomes and quality of life.
Retinitis Pigmentosa Epidemiology Segmentation
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Retinitis Pigmentosa Treatment Landscape
With only one approved therapies like LUXURNA, prompting exploration of off-label and symptomatic treatments, managing this progressive condition involves best supportive care, genetic counseling, and adaptive strategies. The potential therapies in the Retinitis Pigmentosa pipeline, including AGTC-501, Botaretigene sparoparvovec, MCO-010, GS030, ADX-2191, jCell, EA-2353, and others, are advancing through different stages of clinical development, offering promising avenues for effective Retinitis Pigmentosa treatments.
Retinitis Pigmentosa Marketed Drugs
LUXTURNA: Sparks Therapeutics (Roche)/Novartis
LUXTURNA (AAV2-hRPE65v2; voretigene neparvovec), known as voretigene neparvovec-rzyl, is a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. It provides a copy of the RPE65 gene to act in place of the mutated RPE65 gene. This working gene can restore vision and improve sight. The drug is administered as a subretinal single injection below the retina in patients who have confirmed RPE65 mutations and viable retinal cells. The drug was developed and commercialized in the US by Spark Therapeutics. In Europe, Novartis is currently marketing LUXTURNA as per a licensing agreement covering the development, registration, and commercialization rights of LUXTURNA in markets outside the US.
Retinitis Pigmentosa Emerging Drugs
Botaretigene sparoparvovec: Johnson & Johnson Innovative Medicine /MeiraGTx
Botaretigene sparoparvovec (bota-vec) is designed to treat the most common form of X-linked retinitis pigmentosa (XLRP) caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15 (RPGR ORF15). Both rods and cones photoreceptors require RPGR ORF15 to function.The Phase I/II clinical trial of bota-vec in adult and pediatric patients is complete, and the Phase III Lumeos clinical trial completed enrollment in 2023. AAV-RPGR has received Fast Track and Orphan Drug designations from the FDA, as well as PRIME, ATMP, and Orphan Medicinal Product designations from the EMA. Currently, the drug is in the Phase III stage of its development for the treatment of X-linked Retinitis Pigmentosa.
MCO-010: Nanoscope Therapeutics
Nanoscope’s MCO-010 gene therapy utilizes a convenient and well-established intraocular injection for delivery of a gene that encodes for the ambient light-sensitive MCO protein into retinal cells. These therapies are intended to enable retinal cells to detect light so that patients with retinitis pigmentosa or Stargardt disease may see again. MCO-010, is in clinical development for retinitis pigmentosa (RP) and Stargardt disease, which are two rare retinal diseases that cause blindness. MCO-010, recently reported topline results from the RESTORE Phase IIb multicenter, randomized, double-masked, sham-controlled clinical trial in the US for retinitis pigmentosa. The company has also recently completed the Phase II STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease. MCO-010 has received FDA fast track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease.
Retinitis Pigmentosa Drug Market
Optogenetics presents an innovative gene therapy overcoming the limitations of traditional approaches. It operates independently of specific genes and proves effective in late-stage diseases characterized by substantial photoreceptor loss. Retinitis pigmentosa is basically an inherited disease leading to a degeneration of the photoreceptor cells, disrupting the normal physiology of phototransduction. This may be autosomal dominant, autosomal recessive, X-linked, or maternally acquired. Mutations in pre-mRNA splicing cause autosomal dominant retinitis pigmentosa. Autosomal recessive RP is caused when two unaffected individuals who are carriers of the same RP-inducing gene in diallelic form can produce offspring with RP. X-linked RP is identified with mutations of six genes most commonly occurring at specific loci in the RPGR and RP2 genes. These multiple mutations are produced, causing the degeneration of photoreceptor cells.
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Retinitis Pigmentosa Market Outlook
As more targetable mutations are discovered, and new targeted Retinitis Pigmentosa drugs are developed, patients and Opthamologists will have an expanding array of Retinitis Pigmentosa treatment options. Given the rapid pace of drug approvals, it is important to pause and ensure sufficient data supports the use of specific agents in the appropriate treatment settings, including adjuvant, consolidation, first-line, or subsequent therapy.
Currently, LUXTURNA (voretigene neparvovec) is the only approved therapy for retinitis pigmentosa and is only authorized for the treatment of a small subpopulation of patients that have the RPE65 mutation. LUXTURNA gene therapy is designed for both adult and pediatric patients experiencing vision loss from Inherited Retinal Disease (IRD). Companies that focus on both adult and pediatric patients are likely to have a larger patient pool. Some key players pursuing this approach include Beacon Therapeutics (AGTC-501), MeiraGTx/Janssen Research & Development (Botaretigene sparoparvovec), 4D Molecular Therapeutics (4D-125), ProQR Therapeutics/Laboratoires Thea (Ultevursen), Coave Therapeutics (CTx-PDE6b), and Ocugen (OCU400).
The Retinitis Pigmentosa market is driven by several key factors, including the rising prevalence of inherited retinal disorders, growing awareness of rare eye diseases, and advancements in genetic testing and diagnostic tools. Increasing investment in Retinitis Pigmentosa research and the emergence of gene and cell therapies have further accelerated market growth. Moreover, the expanding pipeline of Retinitis Pigmentosa clinical trials and orphan drug designations are attracting interest from pharmaceutical companies. However, the market faces significant barriers such as high costs of advanced treatments, limited availability of curative therapies, and challenges in early diagnosis due to disease heterogeneity. Additionally, the complexity of gene therapy development and stringent regulatory approvals can delay product launches, hindering the overall progress of the Retinitis Pigmentosa treatment market.
Retinitis Pigmentosa Market Dynamics
The dynamics of the retinitis pigmentosa market are expected to change in the coming years. Gene therapy, available in the market, is utilized to address the effects of defective, disease-causing genes by employing engineered viruses, or viral vectors, to deliver a functional gene version into cells. The rising prevalence of retinitis pigmentosa worldwide has spurred pharmaceutical companies to explore this market, aiming for enhanced revenues through specific research and development strategies.
Scope of the Retinitis Pigmentosa Market Report
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Table of Content
1. Key Insights
2. Report Introduction
3. Executive Summary of Retinitis Pigmentosa (RP)
4. Retinitis Pigmentosa Market Overview at a Glance
5. Key Events
6. Epidemiology and Market Forecast Methodology
7. Retinitis Pigmentosa: Disease Background and Overview
8. Treatment of Retinitis Pigmentosa
9. Retinitis Pigmentosa Epidemiology and Patient Population
10. Patient Journey
11. Retinitis Pigmentosa Marketed Drugs
12. Retinitis Pigmentosa Emerging Drugs
13. Retinitis Pigmentosa: Market Analysis
14. Retinitis Pigmentosa Unmet Needs
15. Retinitis Pigmentosa SWOT Analysis
16. Retinitis Pigmentosa KOL Views
17. Retinitis Pigmentosa Market Access and Reimbursement
18. Appendix
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
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