Retinitis Pigmentosa – Opportunity Assessment and Forecast

Retinitis pigmentosa (RP) is an inherited retinal dystrophy that is caused by mutations in genes that code for proteins involved in maintenance and function of the photoreceptor cells, cones and rods. While RP genotypes are heterogenous, it is also important to note that patients with the same mutation can exhibit different phenotypic manifestations. The genetic mutations can cause a number of effects including an inability to produce the required protein, the production of a toxic protein, or the formation of an abnormal protein that doesn’t function properly. Multiple genes have been found to cause RP. It is understood that more than 100 genetic loci on 50 different genes can cause multiple patterns of inheritance and expression for RP. Different types of RP inheritance are identified including autosomal recessive, autosomal dominant, X-linked recessive, and sporadic inheritance.

Spark Therapeutics’ and Roche’s Luxturna (voretigene neparvovec) is the only approved treatment modality that is currently available and is indicated for patients with RP that is associated with biallelic RPE65 mutation. This gene therapy delivers a normal copy of the RPE65 gene to the retinal pigment epithelium (RPE) cells that are lacking a normal functioning RPE65 gene.

Key Questions Answered

– How will the retinitis pigmentosa market landscape in the 3MM (US, Germany, UK) change from 2020-2030?

– What are the most promising late-stage pipeline products for retinitis pigmentosa?

– How do the clinical and commercial attributes of late-stage pipeline products compare with one another, and against existing treatment options?

– What are the remaining unmet needs in retinitis pigmentosa?

– What drivers and barriers will affect retinitis pigmentosa treatment sales in the 3MM over the forecast period?


Overview of retinitis pigmentosa, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and current management strategies.

Topline retinitis pigmentosa market revenue from 2020-2030. Annual cost of treatment and major pipeline product sales in this forecast period are included.

Key topics covered include current treatment options, unmet needs and opportunities, and the drivers and barriers affecting retinitis pigmentosa treatment sales in the 3MM.

Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, synopses of innovative early-stage projects, and detailed analysis of late-stage pipeline products.

Analysis of the current and future market competition in the global retinitis pigmentosa market. Insightful review of the key industry drivers, constraints, and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Key Highlights

During the 10-year forecast period, there is one major pipeline product on track to launch, driving a forecast growth in the 3MM from less than $50m in 2020 to $2 in 2030.

Environmental unmet needs include consistent genetic testing to help identify patients’ underlying retinitis pigmentosa mutations. To combat the unmet need of therapies to help a wider patient population, Biogen’s BIIB112 (cotoretigene toliparvovec) is projected to launch in 2022 in the US and in 2024 in Germany and the UK. Biogen’s BIIB12 is expected to treat a significantly larger subset of retinitis pigmentosa patients than Luxturna, and, in combination with the premium price associated with this gene therapy, driving this market’s potential to over $2B.

However, as gene therapies, pricing for retinitis pigmentosa therapies are very expensive, perhaps prohibitively so depending on the country and payer.

Reasons to buy

The report will enable you to:

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

Develop business strategies by understanding the trends shaping and driving the global retinitis pigmentosa market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the retinitis pigmentosa market in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Companies mentioned

Spark Therapeutics

F. Hoffmann-La Roche



Table of Contents

1 Executive Summary

1.1 Executive Summary

2 Disease Overview

2.1 Etiology and Pathophysiology

3 Epidemiology

3.1 Diagnosed Prevalent Cases of RP, 2020-2030

3.2 Diagnosed Prevalent Cases of RP by Specific Mutation, 2020

4 Current Treatment Options

4.1 Treatment Paradigm

4.2 Marketed Products

4.3 Product Profile: Spark Therapeutics’ Luxturna

5 Unmet Needs and Opportunities

5.1 Unmet Needs in RP

5.2 Streamlining a Holistic Framework for Genetic Testing

5.3 Therapies to Help a Wider Patient Population

6 Pipeline Assessment

6.1 Retinitis Pigmentosa Pipeline Overview

6.2 Leading Pipeline Agents

6.3 Product profile: BIIB-112

7 R&D Strategies

7.1 Trends in Clinical Trial Design in Retinitis Pigmentosa

7.2 Trends in Deal-Making in Retinitis Pigmentosa

8 Market Outlook

8.1 Retinitis Pigmentosa Market Forecast

8.2 Market Drivers and Barriers

9 Appendix

11.1 Primary Research: KOL Information

11.2 Bibliography

11.3 About the Authors

10 Contact Us


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