Erythropoietic Protoporphyria Market

Key Highlights

 

• As per a study by Licha et al. (2021), erythropoietic protoporphyria has been reported worldwide, with prevalence between 1:75,000 and 1:200,000. It usually manifests in early infancy upon the first sun exposures. It is characterised by cutaneous manifestations of acute painful photosensitivity with erythema and oedema, sometimes with petechiae.

 

• The erythropoietic protoporphyria epidemiology covered in the report provides historical as well as forecasted erythropoietic protoporphyria epidemiology [segmented as Total Prevalent cases of Erythropoietic Protoporphyria, Total Diagnosed cases of Erythropoietic Protoporphyria, and Total Treated cases of Erythropoietic Protoporphyria] in the 7MM covering the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2019 to 2032.

 

Request for unlocking the CAGR of Erythropoietic Protoporphyria Market

 

DelveInsight’s “Erythropoietic Protoporphyria – Market Insights, Epidemiology, and Market Forecast – 2032” report delivers an in-depth understanding of the erythropoietic protoporphyria, historical and forecasted epidemiology as well as the erythropoietic protoporphyria market trends in the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan.

 

The erythropoietic protoporphyria market report provides current treatment practices, emerging drugs, erythropoietic protoporphyria market share of the individual therapies, and current and forecasted market size from 2019 to 2032, segmented by seven major markets. The report also covers current erythropoietic protoporphyria treatment practices/algorithms and Erythropoietic Protoporphyria unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

 

Geography Covered

• The United States
• EU4 (Germany, France, Italy, Spain) and the United Kingdom 
• Japan

Study period: 2019–2032

 

Erythropoietic Protoporphyria Market Disease: Understanding and Treatment Algorithm

The DelveInsight’s erythropoietic protoporphyria market report gives a thorough understanding of erythropoietic protoporphyria by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis, and treatment. Erythropoietic protoporphyria is a rare inherited metabolic disorder caused by a deficiency of the enzyme ferrochelatase (FECH), which results from changes (mutations) in the FECH gene. Due to abnormally low levels of this enzyme, excessive amounts of protoporphyrin accumulate in the bone marrow, blood plasma, and red blood cells. Some patients with symptoms of erythropoietic protoporphyria have a genetic change in a different gene called ALAS2. When a patient has a genetic change in this gene, the condition is referred to as X-linked protoporphyria.

 

The major symptoms of these disorders is severe pain on exposure to sunlight and some types of artificial light, such as fluorescent lights (phototoxicity). On sun exposure, patients may first experience tingling, itching, burning of the skin. After continued exposure to light, the skin may become red and swollen. The hands, arms, and face are the most commonly affected areas. Some people with erythropoietic protoporphyria / X-linked protoporphyria may also have complications related to liver and gallbladder function. The most common symptom of erythropoietic protoporphyria and X-linked protoporphyria is severe pain on sun exposure. Some patients may also be sensitive to some types of artificial light. When the skin is exposed to sun, patients first develop tingling, itching, and/or burning of the skin. These symptoms serve as warning signs as longer exposure can result in severe pain. Affected individuals may also have an abnormal accumulation of body fluid under affected areas (edema) and/or persistent redness or inflammation of the skin (erythema).

 

Erythropoietic protoporphyria Diagnosis

The diagnosis of erythropoietic protoporphyria and X-linked protoporphyria may be made by a thorough clinical evaluation, and specialized laboratory tests. Erythropoietic protoporphyria and X-linked protoporphyria are usually diagnosed during infancy or early childhood, due to characteristic symptoms, and by testing the red blood cells (erythrocytes) for increased levels of protoporphyrin. Genetic testing is useful to confirm the diagnosis and identify if it is erythropoietic protoporphyria or X-linked protoporphyria. This information is useful for genetic counseling and testing family members as both are inherited in a different manner. 

 

Erythropoietic protoporphyria Treatment

In erythropoietic protoporphyria, a high potency form of LUMITENE (oral beta-carotene) has been used to improve an affected individual’s tolerance of sunlight. While some patients report improvement, recent studies show that there is no data to support the benefit of this treatment. In 2019, the Food and Drug Administration (FDA) approved SCENESSE (afamelanotide) for the treatment of adult patients with erythropoietic protoporphyria. SCENESSE is an injectable implant and works by increasing skin pigmentation which provides protection and improves sun tolerance.

 

Erythropoietic Protoporphyria Epidemiology 

The erythropoietic protoporphyria epidemiology section provides insights into historical and current erythropoietic protoporphyria patient pool and forecasted trends for seven individual major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the erythropoietic protoporphyria report also provides the diagnosed patient pool, their trends and assumptions undertaken.

 

Key findings

 

• As per a study by Licha et al. (2021), erythropoietic protoporphyria has been reported worldwide, with prevalence between 1:75,000 and 1:200,000. It usually manifests in early infancy upon the first sun exposures. It is characterised by cutaneous manifestations of acute painful photosensitivity with erythema and oedema, sometimes with petechiae. 

 

• The erythropoietic protoporphyria epidemiology covered in the report provides historical as well as forecasted erythropoietic protoporphyria epidemiology [segmented as Total Prevalent cases of Erythropoietic Protoporphyria, Total Diagnosed cases of Erythropoietic Protoporphyria, and Total Treated cases of Erythropoietic Protoporphyria] in the 7MM covering the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2019 to 2032.

 

Country-wise Erythropoietic Protoporphyria Epidemiology

The epidemiology segment also provides the erythropoietic protoporphyria epidemiology data and findings across the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan.

Erythropoietic Protoporphyria Recent Developments

• In January 2025, Disc Medicine received positive feedback from its Type C meeting with the FDA regarding bitopertin for treating Erythropoietic Protoporphyria (EPP). The company plans to submit a New Drug Application (NDA) in H2 2025 under the accelerated approval pathway, using protoporphyrin IX (PPIX) reduction as a surrogate endpoint.

Erythropoietic Protoporphyria Drug Chapters

The drug chapter segment of the erythropoietic protoporphyria report encloses the detailed analysis of erythropoietic protoporphyria marketed drugs and late-stage (Phase III and Phase II) erythropoietic protoporphyria pipeline drugs. It also helps understand the erythropoietic protoporphyria 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.

 

Erythropoietic Protoporphyria Marketed Drugs

 

SCENESSE (afamelanotide): Clinuvel Inc.

Afamelanotide, a synthetic analog of alpha-melanocyte-stimulating hormone (MSH), normally increases the skin pigmentation and decreases the production of cytokines and free radicals. It is given subcutaneously as a controlled released implant (16mg), especially in the summer season. 

 

Note: Detailed current therapies assessment will be provided in the full report of erythropoietic protoporphyria.

 

Erythropoietic Protoporphyria Emerging drugs

The report details the emerging erythropoietic protoporphyria therapies under the late and mid-stage of development for erythropoietic protoporphyria treatment.

 

MT-7117: Mitsubishi Tanabe Pharma

MT-7117 is a novel synthetic, orally-administered, non-peptide small molecule, which acts as a selective agonist of melanocortin-1 receptor (MC1R) with a potential for being effective in the prevention of photo toxicity in erythropoietic protoporphyria (EPP) patients 

 

Note: Detailed emerging therapies assessment will be provided in the full report of erythropoietic protoporphyria.

 

Erythropoietic Protoporphyria Market Outlook

The erythropoietic protoporphyria market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Erythropoietic protoporphyria market trends by analyzing the impact of current therapies on the market, Erythropoietic Protoporphyria unmet needs, and demand for better technology.

 

This segment gives a thorough detail of the erythropoietic protoporphyria market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need for 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.

 

Key player such as Mitsubishi Tanabe Pharma along with few others are involved in developing therapies for erythropoietic protoporphyria.

 

According to DelveInsight, the erythropoietic protoporphyria market in 7MM is expected to witness a major change in the study period 2019–2032.

 

Key findings

This section includes a glimpse of the erythropoietic protoporphyria market in 7MM.

 

The United States: Erythropoietic Protoporphyria Market Outlook

This section provides the total erythropoietic protoporphyria market size and market size by therapies in the United States.

 

EU4 and the UK: Erythropoietic Protoporphyria Market Outlook

The total erythropoietic protoporphyria market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

 

Japan: Erythropoietic Protoporphyria Market Outlook

The total erythropoietic protoporphyria market size and market size by therapies in Japan is also mentioned.

 

Analyst Commentary

• The pipeline of lumbosacral radicular pain is very robust, many potential therapies are being investigated for the treatment of erythropoietic protoporphyria, and it is safe to predict that the treatment space will experience a significant impact on the market during the forecast period.
• The expected introduction of emerging therapies with improved efficacy, more awareness initiatives programs, and further improvement in the diagnosis rate, are likely to boost the growth of the erythropoietic protoporphyria in the 7MM. Aside from that, the market size of erythropoietic protoporphyria may flourish due to increased research and development, label-expansion of approved therapies into other epilepsy in this field.
• The market growth of erythropoietic protoporphyria may offset by failures and/or discontinuation of the emerging therapies, unaffordable pricing, market access and reimbursement issues, and a scarcity of healthcare specialists

 

Erythropoietic Protoporphyria Drug Uptake

This section focuses on the rate of uptake of the potential drugs recently launched in the erythropoietic protoporphyria market or expected to get launched in the market during the study period 2019–2032. The analysis covers erythropoietic protoporphyria market uptake by drugs, patient uptake by therapies, and sales of each drug. 

 

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

 

Erythropoietic Protoporphyria Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses erythropoietic protoporphyria key players involved in developing targeted therapeutics.

 

Erythropoietic protoporphyria Clinical Trial development activities

The Erythropoietic protoporphyria clinical trial report covers detailed information on collaborations, acquisitions, and mergers, licensing patent details, and other information for erythropoietic protoporphyria emerging therapies.

 

Erythropoietic Protoporphyria Reimbursement Scenario

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a 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 epidemiology and market trends, we take KOLs and SMEs' opinions working in the erythropoietic protoporphyria 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 and treatment patterns along with erythropoietic protoporphyria market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

 

Competitive Intelligence Analysis

We perform competitive and market intelligence analysis of the erythropoietic protoporphyria 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.

 

Erythropoietic Protoporphyria Market Report Scope

• Descriptive overview of erythropoietic protoporphyria, disease overview, patient journeys, treatment algorithms, diagnosis, and currently available therapies
• Comprehensive insight into the erythropoietic protoporphyria epidemiology and forecasts in the 7MM
• An all-inclusive account of both the current and emerging therapies for erythropoietic protoporphyria, along with the assessment of new therapies, expected to have an impact on the current treatment landscape
• Exhaustive analysis of the erythropoietic protoporphyria market; historical and forecasted covering drug outreach in the 7MM
• Trends shaping and driving the global erythropoietic protoporphyria market

 

Erythropoietic Protoporphyria Market Report Highlights

• In the coming years, the erythropoietic protoporphyria market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
• The companies and academics are working to assess challenges and seek opportunities that could influence erythropoietic protoporphyria R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition 
• Major players are involved in developing erythropoietic protoporphyria therapies. The launch of emerging therapies will significantly impact the erythropoietic protoporphyria market
• A better understanding of erythropoietic protoporphyria pathogenesis will also contribute to the development of novel therapeutics for erythropoietic protoporphyria
• Our in-depth analysis of the erythropoietic protoporphyria pipeline assets across different stages of development (Phase III and Phase II), emerging trends, and comparative analysis of pipeline products with detailed erythropoietic protoporphyria clinical trial profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

 

Erythropoietic Protoporphyria Report Insights

• Patient-Based Erythropoietic Protoporphyria Market Forecasting
• Therapeutic approaches
• Erythropoietic protoporphyria pipeline analysis
• Erythropoietic protoporphyria market size and trends
• Erythropoietic protoporphyria market opportunities
• Impact of upcoming therapies

 

Erythropoietic Protoporphyria Report Key Strengths

• 11 years forecast
• 7MM coverage
• Erythropoietic protoporphyria epidemiology segmentation
• Key cross competition
• KOL views
• Erythropoietic protoporphyria drugs uptake

 

Erythropoietic Protoporphyria Report Assessment

• Current treatment practices
• Erythropoietic Protoporphyria Unmet needs
• Erythropoietic protoporphyria pipeline product profiles
• Erythropoietic protoporphyria market attractiveness

 

Key Questions

Erythropoietic Protoporphyria Market Insights:

• What would be the erythropoietic protoporphyria market growth till 2032, and what will be the resultant market size in 2032?
•  What was the erythropoietic protoporphyria drug class share (in percentage) distribution in 2019, and how would it look in 2032?
• What would be the erythropoietic protoporphyria total market size and market size by therapies across the 7MM during the forecast period (2019–2032)?
• What are the key findings of the market across 7MM, and which country will have the largest erythropoietic protoporphyria market size during the forecast period (2019–2032)
• How would the unmet needs affect the erythropoietic protoporphyria market dynamics and subsequent analysis of the associated trends?

 

Erythropoietic Protoporphyria Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of erythropoietic protoporphyria?
• What is the historical and forecasted erythropoietic protoporphyria patient pool in 7MM, and where can one observe the highest patient population and growth opportunities? 
• What are the key factors driving the epidemiology trends for seven major markets covering the United States, EU4 (Germany, France, Italy, Spain) and the United Kingdom, and Japan?

 

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current treatment guidelines and treatment options, in addition to approved therapies for erythropoietic protoporphyria in the US, Europe, and Japan?
• What are the key collaborations (Industry–Industry, Industry-Academia), mergers and acquisitions, and licensing activities related to erythropoietic protoporphyria therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for erythropoietic protoporphyria and its status, along with the challenges faced?

 

Reasons to Buy

• The Patient-Based Erythropoietic Protoporphyria Market Forecasting report will help in developing business strategies by understanding trends shaping and driving the erythropoietic protoporphyria market
• Organize sales and marketing efforts by identifying the best opportunities for erythropoietic protoporphyria in the US, EU4 (Germany, France, Italy, Spain) and the UK, and Japan
• Identification of strong upcoming players in the market that will help devise strategies that will help in getting ahead of competitors