This report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the market trends of Retinitis Pigmentosa (RP) in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Diagnosed Prevalent Cases of Retinitis Pigmentosa in the 7MM, Gender-Specific Diagnosed Prevalence of Retinitis Pigmentosa in the 7MM, Type-Specific Diagnosed Prevalence of Retinitis Pigmentosa in the 7MM, Subtype-Specific Diagnosed Prevalence of Syndromic and Systemic RP in the 7MM, and Subtype-specific Diagnosed Prevalent Cases of Non-Syndromic RP in the 7MM) scenario of Retinitis Pigmentosa in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017-2030.
This section provides glimpses of the Retinitis Pigmentosa epidemiology in the 7MM market. According to the report, the total diagnosed prevalent population of Retinitis Pigmentosa (RP) in the 7MM was 253,420 in 2017. These cases are expected to increase with a significant CAGR during the study period (2017-2030).
The epidemiology segment also provides the Retinitis Pigmentosa epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The drug chapter segment of the Retinitis Pigmentosa report encloses the detailed analysis of Retinitis Pigmentosa marketed drugs and late stage (phase III) pipeline drugs. It also helps to understand the RP clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.
Luxturna: Spark Therapeutics/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. It is indicated for the treatment of patients with vision loss due to Leber's congenital amaurosis or retinitis pigmentosa inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations. Emerging Drugs
Horama is developing a HORA-RPE65 gene replacement product for the treatment of RPE65 Retinitis Pigmentosa. It is an AAV 2/4 encapsidating the human RPE65 gene under the control of the human native RPE65 promoter. The company investigational candidate HORA-RPE65 provides the cell with a non-mutated copy of the human RPE65 gene, which can express functional RPE65 protein to halt or at least significantly delay retinal degeneration in patients with inherited retinal dystrophies caused by RPE65 gene mutations.
The drug is being delivered in the form of a sterile vector suspension injected directly into the subretinal space, where it induces transgene expression in retinal pigment epithelium cells. Horama had already completed a single-center, open, non-randomized phase I/II clinical trial which accessed the safety and effectiveness of HORA-RPE65 in patients with retinal dystrophy caused by RPE65 gene abnormalities.
The Retinitis Pigmentosa market outlook of the report helps to build a detailed comprehension of the historic, current and forecasted Retinitis Pigmentosa market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand of better technology.
This segment gives a thorough detail of Retinitis Pigmentosa market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to the report, Retinitis Pigmentosa 7MM market is expected to change in the study period 2017-2030.
This section includes a glimpse of the Retinitis Pigmentosa 7MM market. The market size of Retinitis Pigmentosa in the 7MM market was USD 237.27 million in 2017.
The United States Market Outlook
This section provides the total Retinitis Pigmentosa market size and market size by therapies in the United States.
Among the 7MM countries, the United States had the highest market size of RP in 2017, which accounts for 56.40% of the total market. At present, the growth of market size for RP is attributed to support treatment regimens, the only approved therapy (Luxturna), as well as, emerging therapies. The market size for RP was found to be USD 133.82 million in 2017.
EU-5 Market Outlook
The total Retinitis Pigmentosa market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.
Germany accounts for 26.28% of the total EU-5 market of RP. The publisher's estimates suggest that the overall market size of RP was observed to remain nearly constant from USD 20.90 million in 2017 to USD 21.33 million in 2018. This is attributed to the dependency of the market size of RP on supportive treatment regimens, at present. However, with the recent approval of Luxturna, the market size of RP is expected to experience a significant increase. The dearth of marketed treatment regimens for RP is the foremost unmet need of this therapeutic area, which is primarily responsible for nearly constant market size.
Japan Market Outlook
The total Retinitis Pigmentosa market size and market size by therapies in Japan are provided.
This section focusses on the rate of uptake of the potential drugs recently launched in the Retinitis Pigmentosa market or expected to get launched in the market during the study period 2017-2030. The analysis covers Retinitis Pigmentosa market uptake by drugs; patient uptake by therapies; and sales of each drug.
The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing and patent details for Retinitis Pigmentosa emerging therapies.
Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.
KOL - Views
To keep up with current market trends, we take KOLs and SME's opinion working in Retinitis Pigmentosa domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Retinitis Pigmentosa market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.
Competitive Intelligence Analysis
The publisher performs competitive and market Intelligence analysis of the Retinitis Pigmentosa market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.