Retinitis pigmentosa (RP) is a rare genetic disorder marked by retinal degeneration, progressive vision loss, and eventual blindness. There are no approved pharmacotherapies for RP, and current disease management options comprise nutritional supplementation (e.g., vitamin A, omega-3 fatty acids), off-label treatments for comorbidities (e.g., carbonic anhydrase inhibitors), and approved retinal prosthetic devices (i.e., Second Sight’s Argus II, Retinal Implant AG’s Alpha IMS). Significant unmet clinical need and commercial opportunity exists for medications with disease-modifying potential. The RP clinical pipeline is robust and diverse, comprising gene therapy, stem-cell therapy, and other approaches such as neuroprotection and stimulation of neurogenesis. However, the majority of these therapies are in early phases of development and face many scientific, clinical, and developmental hurdles in order to obtain regulatory approval for the treatment of RP.

Questions Answered

How will the size of the RP population change through 2026? What percentage of the RP population receives drug treatment?

What are the key drug targets and technologies emerging from basic and clinical research in RP? Which emerging therapies have RP experts intrigued or optimistic? How would new therapies influence management of RP patients?

The RP pipeline comprises various mechanisms of action and technologies. How are these therapies being evaluated, and which are likely to launch by 2026? What commercial impact will they have on the RP market?

Product Description

Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Table of contents

  • Retinitis Pigmentosa - Landscape & Forecast - Disease Landscape & Forecast
    • Executive Summary
      • Key Findings
        • Regional Sales of Key Therapies to Treat Retinitis Pigmentosa: 2026
        • Retinitis Pigmentosa SWOT Analysis
        • Actionable Recommendations in Retinitis Pigmentosa According to Ophthalmologists
    • Commercial Outlook
      • Key Findings
        • Expert Insight
      • Drivers and Constraints
        • What Factors Are Driving Sales in Retinitis Pigmentosa?
        • What Factors Are Constraining Sales in Retinitis Pigmentosa?
      • Drug-Class-Specific Trends
        • Gene Therapy
        • Retinoid Replacement Therapy
      • Alternative Market Scenarios
        • Alternative Scenarios for the Retinitis Pigmentosa Market Through 2026
    • Forecast
      • Sales of Key Therapies for Retinitis Pigmentosa in 2026
    • Etiology and Pathophysiology
      • Key Findings
        • Disease Overview
      • Etiology
        • Etiology of Retinitis Pigmentosa
        • Major Genes Involved in the Etiology of Retinitis Pigmentosa
      • Pathophysiology
        • Pathophysiology of Retinitis Pigmentosa
      • Disease Classification
        • Signs and Symptoms of Retinitis Pigmentosa
          • Key Pathways and Drug Targets
          • Epidemiology Overview
            • Key Findings
              • Epidemiology Populations
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Retinitis Pigmentosa
                • Number of Diagnosed Prevalent Cases of Retinitis Pigmentosa in the Major Pharmaceutical Markets, 2016-2026
                • Number of Drug-Treated Prevalent Cases of Retinitis Pigmentosa in the Major Pharmaceutical Markets, 2016-2026
                • Disease Definition
                • Methods
                • Sources Used for Clinical Subtypes of Retinitis Pigmentosa
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Usher Syndrome
                • Number of Diagnosed Prevalent Cases of Usher Syndrome in the Major Pharmaceutical Markets, 2016-2026
                • Disease Definition
                • Methods
                • Sources Used for Prevalence of RPE65 Mutation
                • Number of Diagnosed Prevalent Cases of RPE65 Mutation in the Major Pharmaceutical Markets, 2016-2026
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Leber's Congenital Amaurosis
                • Number of Diagnosed Prevalent Cases of Leber's Congenital Amaurosis in the Major Pharmaceutical Markets, 2016-2026
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Choroideremia
                • Number of Diagnosed Prevalent Cases of Choroideremia in the Major Pharmaceutical Markets, 2016-2026
            • Current Treatment Overview
              • Key Findings
                • Expert Insight
              • Diagnosis
                • Diagnostic Tests for Retinitis Pigmentosa
                • Treatment Providers and Referral Patterns
                • Expert Insight: Diagnosis of Retinitis Pigmentosa
              • Treatment Goals
                • Key End Points Used in Clinical Trials for Retinitis Pigmentosa
                • Physician Insights on Clinical End Points
                • Expert Insight: Clinical End Points
              • Key Current Therapies
                • Overview
                • Mechanism of Action of Key Current Drug Classes Used for Retinitis Pigmentosa
                • Current Treatments Used for Retinitis Pigmentosa
                • Market Events Impacting the Use of Key Current Therapies in Retinitis Pigmentosa
                • Clinical Analysis of Argus II for the Treatment of Retinitis Pigmentosa
                • Ongoing Clinical Development
                • Advantages and Disadvantages of Argus II
                • Expert Insight: Argus II
                • Clinical Analysis of Alpha IMS for the Treatment of Retinitis Pigmentosa
                • Ongoing Clinical Development
                • Advantages and Disadvantages of Alpha IMS
                • Expert Insight: Alpha IMS
                • Select Clinical Trial Outcomes for Carbonic Anhydrase Inhibitors
                • Advantages and Disadvantages of Carbonic Anhydrase Inhibitors
                • Expert Insight: Carbonic Anhydrase Inhibitors
              • Medical Practice
                • Overview
                • Retinitis Pigmentosa Treatment Guidelines
                • Generalized Treatment Decision Tree for Retinitis Pigmentosa
            • Unmet Need Overview
              • Key Findings
                • Expert Insight
              • Attainment of Unmet Needs
                • Current Attainment of Unmet Needs in Retinitis Pigmentosa
                • Future Attainment of Unmet Needs in Retinitis Pigmentosa
                • Top Unmet Needs in Retinitis Pigmentosa: Current and Future Attainment
            • Emerging Therapies Overview
              • Key Findings
                • Expert Insight
                • Pipeline Trends for Retinitis Pigmentosa
              • Key Emerging Therapies
                • Notable Developments Among Key Emerging Therapies for Retinitis Pigmentosa
                • Key Emerging Therapies in Development for Retinitis Pigmentosa
                • Key Results from Select Clinical Trials Investigating Luxturna for the Treatment of Retinitis Pigmentosa
                • Luxturna Clinical Development
                • Expert Insight on Use of Luxturna in the Treatment of Retinitis Pigmentosa
                • Expert Insight: Luxturna
                • Expectations for Launch and Sales Opportunity of Luxturna in Retinitis Pigmentosa
                • Key Ongoing Clinical Trials of RST-001 in the Treatment of Retinitis Pigmentosa
                • Expert Insight on Use of RST-001 in the Treatment of Retinitis Pigmentosa
                • Expert Insight: RST-001
                • Expectations for Launch and Sales Opportunity of RST-001 in Retinitis Pigmentosa
                • Key Results from Select Clinical Trials Investigating Zuretinol Acetate for the Treatment of Retinitis Pigmentosa
                • Zuretinol Acetate Clinical Development
                • Expert Insight on Use of Zuretinol Acetate in the Treatment of Retinitis Pigmentosa
                • Expectations for Launch and Sales Opportunity of Zuretinol Acetate in Retinitis Pigmentosa
                • Key Results from Select Clinical Trials Investigating NT-501 for the Treatment of Retinitis Pigmentosa
                • NT-501 Clinical Development
                • Expert Insight on Use of NT-501 in the Treatment of Retinitis Pigmentosa
                • Expectations for Launch and Sales Opportunity of NT-501 in Retinitis Pigmentosa
              • Early-Phase Pipeline Analysis
                • Notable Developments in the Early-Phase Pipeline for Retinitis Pigmentosa
                • Select Therapies in Early Clinical Development for Retinitis Pigmentosa
                • Select Therapies in Preclinical Development for Retinitis Pigmentosa
              • Patient Registries
                • Patient Registries for Retinitis Pigmentosa
                • Prominent Retinitis Pigmentosa Patient Organizations
              • Orphan Drug Designation
              • Access & Reimbursement Overview
                • Region-Specific Reimbursement Practices
                • Methodology
                  • Bottom-Up Forecasting Overview
                    • Patient Populations
                    • Drug-Specific Assumptions
                  • Bottom-Up Forecast Assumptions
                    • General Sources of Data
                    • Drug Treatment Rate Assumptions in Retinitis Pigmentosa
                    • Patient Share Assumptions in Retinitis Pigmentosa
                    • General Statements About Pricing
                    • Sources for Drug Prices
                    • 2016 Exchange Rates
                    • Price per Procedure or Treated Day: 2026
                    • Dosing, Days of Therapy, and Compliance
                    • Out-Year Forecasting
                    • Emerging Therapy Prices
                  • Primary Market Research
                    • Experts Interviewed
                • Appendix
                  • Key Abbreviations Related to Retinitis Pigmentosa
                  • Brands, Marketers, and Generic Availability of Key Therapies for Retinitis Pigmentosa, by Market
                  • Bibliography

              Author(s): Akash Saini, Ph.D.; Vishnu Babykumar, MSc

              Akash is a Principal Analyst in the China In-Depth team at Decision Resources Group. Since 2016, he has specialized in a range of indications including rare disease indications such as DMD, ALS, ITP, IPF, JIA, and retinitis pigmentosa. He has authored Disease Landscape & Forecast reports, Access & Reimbursement reports, and Treatment Algorithm reports based on primary market research and real-world evidence. Prior to joining DRG, Akash was a post-doctoral fellow at the University of Massachusetts Medical School. He has a Ph.D. from the International Centre for Genetic Engineering and Biotechnology, New Delhi.

              Tech savvy, jovial and a research enthusiast; member of the DRG family since April 2015 and working from the India office.

              Started off my career path with a bachelor’s in Biomedical Engineer followed by a master’s in Cardiovascular Sciences; having worked in the I.T industry and as a Junior Epidemiologist at ViS Research prior to DRG.