Retinitis Pigmentosa Market Insights 2034: EMA, PDMA, FDA Approvals, Clinical Trials, Prevalence, Medication, Revenue, Statistics, Pipeline and Companies by DelveInsight

(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
• The total Retinitis Pigmentosa Treatment Market Size in the 7MM was ~USD 500 million in 2023, which is expected to grow significantly during the forecast period 2024-2034.
• The leading Retinitis Pigmentosa Companies such as Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic Sight, jCyte, Endogena Therapeutics, ProQR Therapeutics, and Aldeyra Therapeutics and others.
• Promising Retinitis Pigmentosa Therapies such as Botaretigene sparoparvovec, AGTC-501, GS030, 4D 125, CTx PDE6B, OCU 400, EA-2353, Ultevursen, ADX 2191, and others.
• In March 2025, Researchers with Nanoscope Therapeutics Inc., a biotechnology company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, have announced the publication of a paper, "A synthetic opsin restores vision in patients with severe retinal degeneration," in Molecular Therapy, the flagship journal of the American Society of Gene & Cell Therapy. The publication of the paper marks a major breakthrough in mutation-independent optogenetic monotherapy for patients suffering from inherited retinal diseases.
• In February 2025, The US Food and Drug Administration (FDA) approved an Investigational New Drug (IND) application for a Phase III trial involving an iPSC-based therapy. Meanwhile, the European Medicines Agency (EMA) has granted a positive Advanced Therapy Medicinal Product (ATMP) classification opinion for a gene therapy targeting retinitis pigmentosa. Additionally, the California Institute for Regenerative Medicine (CIRM) has awarded a $6 million grant to researchers at the Keck School of Medicine, University of Southern California (USC), to advance the development of a new genetic therapy for glioblastoma.
• In January 2025, Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company specializing in gene and cell therapies, biologics, and vaccines, has provided a positive two-year safety and efficacy update for its Phase 1/2 OCU400 clinical trial. Designed for treating early to advanced retinitis pigmentosa (RP) in both pediatric and adult patients, OCU400 offers hope to approximately 2 million individuals worldwide, including around 300,000 in the U.S. and EU, who risk legal blindness. This one-time therapy aims to provide long-term benefits to all affected patients.
• In January 2025, Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NPI-001 (N-acetylcysteine amide) tablets, Nacuity's proprietary investigational therapy for the treatment of patients with retinitis pigmentosa (RP).
• In January 2025, Beacon Therapeutics announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for treating X-linked retinitis pigmentosa (XLRP), advancing its mission to restore vision for patients with blinding retinal diseases.
• In January 2025, ViGeneron GmbH, a next-generation clinical-stage gene therapy company, today announced two important milestones for its novel gene therapy candidate VG901, to treat patients with retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to VG901, and the independent Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing Phase 1b clinical trial.
• In August 2024, Ocugen, Inc. ("Ocugen" or the "Company") (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that it has received notification from FDA to begin its expanded access program (EAP) for the treatment of adult patients, aged 18 and older, with retinitis pigmentosa (RP) with OCU400-a modifier gene therapy product candidate.
• June 2024:- Rolfs Consulting und Verwaltungs-GmbH (RCV)- Participants at risk for a syndromic or a monogenic genetic obesity, incl. participants clinically diagnosed with Bardet-Biedl-Syndrome (BBS).
• May 2024:- PYC Therapeutics- The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11.
• May 2024:- Janssen Pharmaceutical- Phase 3 Study to Evaluate the Safety and Efficacy of AAV5-hRKp.RPGR for the Treatment of Japanese X-linked Retinitis Pigmentosa Associated With Pathogenic Variants in Retinitis Pigmentosa GTPase Regulator (RPGR). The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).
• In February 2024, jCyte, Inc., a pioneering biotechnology company dedicated to preserving and restoring vision in patients with retinitis pigmentosa (RP) and other degenerative retinal disorders, is pleased to announce the successful outcome of its pre-phase 3 Type B meeting with the US Food and Drug Administration (FDA) held on January 16, 2024. Additionally, the company is gearing up to commence its pivotal US trial for jCell in the second half of 2024.
• In the US, the total number of prevalent cases of retinitis pigmentosa were ~113,000 in 2023.
• Nonsyndromic retinitis pigmentosa is more prevalent than syndromic retinitis pigmentosa with ~65% cases of total retinitis pigmentosa.
• X-linked retinitis pigmentosa in the US acoounted for ~16,000 prevalent cases in 2023.
• Germany has the most cases among EU4 and UK, whereas the UK has the fewest.
• RPE65-linked IRD account for about 3-16% of Leber congenital amaurosis and approximately 0.6-6% of retinitis pigmentosa.

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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
• Total Retinitis Pigmentosa Diagnosed Prevalent Population
• Retinitis Pigmentosa Gender-Specific Diagnosed Prevalence
• Retinitis Pigmentosa Type-Specific Diagnosed Prevalence
• Sub-Type Specific Diagnosed Prevalence of Syndromic and Systemic Retinitis Pigmentosa
• Sub-Type Specific Diagnosed Prevalence of Non-Syndromic Retinitis Pigmentosa

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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
• Coverage- 7MM
• Retinitis Pigmentosa Companies- Johnson & Johnson Innovative Medicine (NYSE: JNJ), MeiraGTx (NASDAQ: MGTX), Beacon Therapeutics (Private), Nanoscope Therapeutics (NASDAQ: NASC), Gensight Biologics (EPA: SIGHT), 4D Molecular Therapeutics (NASDAQ: FDMT), Coave Therapeutics (Private), Ocugen (NASDAQ: OCGN), Bionic Sight (Private), jCyte (Private), Endogena Therapeutics (Private), ProQR Therapeutics (NASDAQ: PRQR), Aldeyra Therapeutics (NASDAQ: ALDX), and others.
• Retinitis Pigmentosa Therapies- Botaretigene sparoparvovec, AGTC-501, GS030, 4D 125, CTx PDE6B, OCU 400, EA-2353, Ultevursen, ADX 2191, and others.
• Retinitis Pigmentosa Market Dynamics: Retinitis Pigmentosa Market Drivers and Barriers
• Retinitis Pigmentosa Market Access and Reimbursement, Unmet Needs and Future Perspectives

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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

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