Retinitis Pigmentosa Therapeutics – Pipeline Analysis, Epidemiology and Drug Forecasting

Overview

Retinitis pigmentosa (RP) is identified as a group of rare genetic disorders associated with the breakdown and loss of cells in the retina. The commonly occurring symptoms include loss of peripheral or central vision and affected night vision. The symptoms worsen with time and are often reported in patients aged 34 to 64 years.

The patient can be diagnosed with RP with confirmed genetic mutations. In majority of the cases, the disease is associated with a recessive gene, wherein the patient inherits the affected gene from both parents. However, X-linked dominated genes have also been reported in RP patients. On the basis of clinical impact, RP can be further categorized into syndromic, non-syndromic, and systemic. Syndromic RP and systemic RP affect other neurosensory systems, whereas non-syndromic RP does not affect other organs or tissues.

As per the current scenario, the treatment and supportive therapy indicated for RP patients are dependent on the type of RP; however, till date, there is no any standardized treatment available to address all forms of RP. This indicates that there is high unmet medical need with regard to the respective treatments. Currently, Luxturna, developed by Spark Therapeutics Inc. is the only gene therapy that is approved by the United States Food and Drug Administration (USFDA) and European Medicines Agency (EMA), for the treatment of patients with RP, associated with RPE65 gene mutation.

In addition, a number of drug manufacturers are actively involved in the development of RP therapeutics. For instance, HORA-RPE65 by HORAMA S.A. is developing in the late stage of development, and its HORA-PDE6B is in the mid-stage of development. Some other emerging therapies include AAV8-RPGR (Nightstar Therapeutics Limited), jCell (jCyte Inc.), Cenegermin (Dompé farmaceutici S.p.A.), and ReN003 (ReNeuron Group Plc).

Positive clinical trial results and adoption of collaboration strategies are enhancing RP therapeutics pipeline. Moreover, the issuance of patents helps in achieving different milestones in the form of grants and designations from regulatory bodies and institutes, including the USFDA, EMA, and National Institutes of Health (NIH), among others.

Pipeline Analysis

As of March 2020, the RP therapeutics pipeline comprised 18 pipeline drugs in different stages of development along with one marketed product.

Epidemiology Analysis

The report provides epidemiology forecast of RP for 7 major markets (7MMs) that are the U.S., Japan, and EU5 countries (the U.K., Germany, France, Italy, and Spain). It covers prevalent population and treated patient population for the period 2014–2030. Highlights of the epidemiology are as follows:

• The total diagnosed prevalent population of RP in the 7MM was reported to be approximately 269,500 in 2019.
• As per the estimation, the U.S. reported highest diagnosed cases of RP in 2019.
• Japan also reported significant diagnosed prevalent cases of RP in 2019.
• Among the EU5 countries, Germany had the highest diagnosed prevalence of RP, followed by France.
• Majority of the RP cases were found in the male population, across the 7MM countries.
• RP is mostly diagnosed in patients aged between 35 and 64 years.

Market Analysis

The report also provides market analysis of RP therapeutics for aforementioned 7MMs. It covers market size of supportive treatments, approved treatment, that is, Luxturna, and emerging therapies that are expected to be introduced during the forecast period (2020–2030). Based on the analysis conducted, the highest revenue contributor to the RP therapeutics market was U.S. in 2019, in the 7MM. On the other hand, among the EU5 countries, Spain had the highest market share, whereas Italy had the lowest.

Competitive Landscape

Some of the key players involved in the development of RP therapeutics are Spark Therapeutics Inc., HORAMA S.A., Nightstar Therapeutics Limited, jCyte Inc., ReNeuron Group Plc, Dompé Farmaceutici S.p.A., MeiraGTx Holdings plc, Applied Genetics Technologies Corporation, Biogen Inc., and Allergan plc.

Report Insights

Some highlights of the report “Retinitis Pigmentosa Therapeutics – Pipeline Analysis, Epidemiology and Drug Forecasting” are mentioned below:

• Detailed pipeline analysis of therapeutic candidates that are being developed for the treatment of RP, and estimates and analyses of emerging therapies and their progress status in different phases of development
• Comprehensive insights into pipeline products, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences
• Competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with relevance to RP
• Epidemiology forecast of RP for 7MMs, including the U.S., Japan, and EU5 countries
• Analysis of approved and supportive therapy for RP
• Detailed regulatory framework for drug approval in the U.S., Europe, and Japan
• Insights on the unmet medical needs
• Attribute analysis; expected launch timeline of emerging therapies
• Market size of RP; and expected market share of late-stage clinical drugs in 2024
• SWOT analysis
• Key winning strategies and key highlights of the companies involved in the development of RP, followed by Pharma Proff’s views