retinitis pigmentosa market insights epidemiology and market forecast

DelveInsight's 'Retinitis Pigmentosa (RP)- Market Insights, Epidemiology and Market Forecast-2028 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the market trends of RP in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of RP from 2017 to 2028 segmented by the seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.

Geography Covered

• The United States

• EU5 (Germany, France, Italy, Spain and the United Kingdom)

• Japan

Study Period: 2017-2028

Retinitis Pigmentosa (RP) - Disease Understanding and Treatment Algorithm

Retinitis Pigmentosa (RP), which belongs to group of pigmentary retinopathies and generally starts, refers to a group of inherited retinal disorders causing retinal degeneration and blindness. RP is characterized by progressive bilateral degeneration of the rod and cone photoreceptors that leads to night blindness and progressive visual field defects. Symptoms of RP usually develop between the ages of 10 and 30, although some people experience symptoms during childhood. Symptoms vary depending on what part of the retina is affected. RP is a progressive disease, but the rate at which vision deteriorates varies for each person. Eventually, most people with the disease have a very restricted field of vision.

RP is primarily classified based on the clinical impact, as Non-syndromic, or "simple" (not affecting other organs or tissues); Syndromic (affecting other neurosensory systems such as hearing); Systemic (affecting multiple tissues. Further, the non-syndromic RP is classified on the basis of inheritance pattern, i.e. Autosomal dominant RP, Autosomal recessive RP, X-linked RP, Sporadic RP, and Leber's Congential Amaurosis. Moreover, the syndromic RP include the following subtypes: Usher syndrome and Bardet-Biedl syndrome, which are most frequently occurring among all subtypes.

The DelveInsight's RP market report gives the thorough understanding of the RP by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for RP in the US, Europe, and Japan.

Retinitis Pigmentosa (RP) - Epidemiology

The RP epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight's report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology [segmented by Total Diagnosed Prevalent Population, Gender- Specific Diagnosed Prevalence, Type - Specific Diagnosed Prevalence, Sub-Type Specific Diagnosed Prevalence of Syndromic RP and Sub-Type Specific Diagnosed Prevalence of Non-Syndromic RP] scenario of RP in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2017-2028.

As per DelveInsight's analysis, a higher percentage of diagnosed prevalence was observed for males, in comparison to females, in all the 7MM countries, except Japan, wherein females occupy a larger patient pool than males. According to Delveinsight, the diagnosed prevalent population of RP in the 7MM was assessed to be more than 250,000 in 2017, which is expected to grow at a CAGR of XX% for the study period i.e. 2017-2028.

The estimates show highest diagnosed prevalence of RP in the United States, with an estimated 108,787 cases in 2017. Among the European 5 countries, Germany had the highest diagnosed prevalent population of RP, followed by France and the United Kingdom. On the other hand, Spain had the lowest diagnosed cases. Japan witnessed 28,051 prevalent cases for RP in 2017. Based on the type of RP, we have assessed that majority of the patients suffer with non-syndromic RP, while syndromic and systemic RP cases account for nearly one-fourth of the total RP population.

Furthermore, DelveInsight's analysts suggests that among Syndromic and Systemic RP cases, Usher Syndrome and Bardet-Biedl syndrome are the most diagnosed prevalent subtypes of the disease. In contrast, the trend in percentage of patients for subtypes of non-syndromic RP remains variable throughout the 7MM countries. However, in all the 7MM countries, X-linked RP and Leber congenital amaurosis remain the least occurring subtypes of the disease

Retinitis Pigmentosa (RP) - Drug Chapters

This segment of the RP report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the 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.

Horama is developing a gene therapy candidate HORA-RPE65 in Phase III for the treatment of Autosomal Recessive RP (Non-syndromic) RPE65 RP. It is an AAV 2/4 encapsidating the human RPE65 gene under the control of the human native RPE65 promoter. The company's 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.

Nightstar Therapeutics is developing a sub-retinal AAV-based Adeno-Associated Viral Vector Encoding RP GTPase Regulator gene therapy (AAV8-RPGR) in Phase II/III for Non-syndromic X-linked Retinitis Pigmentosa (XLRP). The company's NSR-RPGR gene therapy consists of a standard AAV8 vector, including the codon-optimized human RPGR DNA. The drug candidate has already demonstrated positive outcomes in the initial Phase of Phase II/III XIRIUS trial.

jCyte is evaluating jCell human retinal progenitor cells in a Phase II trial in Adult patients with RP. The therapy uses adult retinal progenitor cells to rescue rods and cones from RP progression and possibly generate new retinal cells. The treatment requires a single intravitreal injection, which can be performed with topical anesthetic. jCyte has already completed a phase I/IIa study to determine the therapy's safety. Furthermore, the drug received US FDA Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designation.

Neurotech is evaluating Renexus (NT-501) in Phase II in Participants with Syndromic RP using rates of change in cone spacing and density. Renexus (NT-501) consists of encapsulated human cells genetically modified to secrete therapeutic doses of a ciliary neurotrophic factor into the back of the eye for the treatment of RP. The drug candidate was granted orphan drug and fast-track designation by the US FDA for RP.

ReNeuron is currently leading a Phase IIa trial for the assessment of proprietary cGMP-produced, Subretinal, allogeneic, cryopreserved human retinal progenitor cells (hrpc). The company received Orphan Drug in EU and US and Fast Track Designation for in the US, for hrpc, for the treatment of RP.

Retinitis Pigmentosa (RP) - Market Outlook

The RP market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market 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 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.

Current treatments for RP are limited, but a number of developments are dignified to enter the therapeutic field. Based on randomized clinical trials with vitamins and supplements, the only widely recommended treatment is supplementation with high-dose vitamin A palmitate and fish oil, along with avoidance of vitamin E, but these adjustments only delay degeneration.

The accessibility of the retina for relatively safe surgical procedures and the immune privilege of the eye have made retinal diseases the ideal setting to use leading-edge tools, such as gene therapy and stem cell therapy, which have the potential to produce an effective treatment for some types of RP. In Gene therapy, the strategy is relatively simple for RP due to loss-of-function (usually recessively inherited).

There are no standard treatments for patients with retinitis pigmentosa. The most widely recommended treatment for many years has been supplementation with vitamin A, which some studies have shown to slow the rate of retinal deterioration.

So far, only one treatment regimen, Luxturna (Spark Therapeutics), has been approved in the United States and Europe (although not yet marketed in European countries). Luxturna, known as voretigene neparvovec-rzyl, is one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. The therapy received approval by the US FDA in December 2017 and by the EMA in November 2018. It provides a working 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 candidate is administered as a subretinal single injection below the retina in patients who have confirmed RPE65 mutations and viable retinal cells. The therapy is indicated for the treatment of patients with vision loss due to Leber's congenital amaurosis or retinitis pigmentosa inherited retinal dystrophy caused by onfirmed biallelic RPE65 mutations.

Retinitis Pigmentosa (RP) - Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

To meet the unmet need of the market and to provide better versions of current treatment practices several companies have shifted their focus toward this therapeutic area, and several therapies are expected to enter the treatment market of RP during our forecast period, including HORA-RPE65 (Horama), AAV8-RPGR (Nightstar Therapeutics), Jcell (jCyte), Renexus (NT-501; Neurotech Pharmaceuticals), and hRPC (ReNeuron Limited). With the launch of the effective branded targeted therapies, the market is going to rise.

RP Report Insights

• Patient Population

• Therapeutic Approaches

• Pipeline Analysis

• Market Size and Trends

• Market Opportunities

• Impact of upcoming Therapies

RP Report Key Strengths

• 10 Years Forecast

• 7MM Coverage

• Epidemiology Segmentation

• Key Cross Competition

• Market Size by Therapies

• Drugs Uptake

RP Report Assessment

• Pipeline Product Profiles

• Key Products and Key Players

• Market Drivers and Barriers

Key Benefits

• This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving RP market

• Organize sales and marketing efforts by identifying the best opportunities for RP market

• To understand the future market competition in the RP market.