Paroxysmal Nocturnal Hemoglobinuria- Market Insight, Epidemiology and Market Forecast -2030

Paroxysmal Nocturnal Hemoglobinuria- Market Insight, Epidemiology and Market Forecast -2030

  • February 2021 •
  • 150 pages •
  • Report ID: 6028034 •
  • Format: PDF
‘Paroxysmal nocturnal hemoglobinuria (PNH)– Market Insights, Epidemiology, and Market Forecast—2030’ report deliver an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of PNH in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
The PNH market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted PNH market Size from 2017 to 2030, segmented by seven major markets. The report also covers the current PNH treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geographies Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2017–2030

Paroxysmal nocturnal hemoglobinuria: Disease Understanding and Treatment Algorithm

Paroxysmal nocturnal hemoglobinuria Overview
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of the pluripotent hematopoietic stem cell; therefore, it can affect erythrocytes, leukocytes, thrombocytes, and probably some endothelial cells. These hematopoietic stem cells have acquired a somatic mutation in an X?linked gene: the phosphatidylinositol glycan class A (PIG?A). This gene is required to synthesize the glycosylphosphatidylinositol (GPI) anchor, which is necessary to attach some proteins to the cell membrane.
The lack of synthesis of the GPI anchor leads to the underexpression of various proteins on the hematopoietic stem cell surface and on all cell lines generated by it. By this mechanism, a lack of two important complement regulatory proteins is observed on the cell surface: ‘decay?accelerating factor’ (DAF), also called ‘CD55’ and ‘membrane inhibitor of reactive lysis’ (MIRL), also called ‘CD59’. Thus, red blood cells are more vulnerable to the action of complement. This leads to complement?mediated intravascular hemolysis. As a result, a high concentration of free hemoglobin is found in the plasma, responsible for nitric oxide (NO) scavenging. NO depletion causes the majority of symptoms experienced by paroxysmal nocturnal hemoglobinuria (PNH) patients. Smooth muscle dystonia is responsible for dysphagia and abdominal pain. Erectile dysfunction is frequent. NO depletion may also contribute to the development of arterial constriction, leading to reduced blood flow to the kidneys (with renal failure), arterial hypertension, and pulmonary hypertension (associated with frequent but underdiagnosed pulmonary embolism).
No universally accepted classification scheme is available. However, the International PNH Interest Group classifies PNH into three categories: classical PNH (in which patients have clinical manifestations of hemolysis or thrombosis); PNH in the context of other primary bone marrow disorders (such as aplastic anemia or myelodysplastic syndromes); and subclinical PNH, in which patients have low proportions of PNH clones but no clinical or laboratory evidence of hemolysis or thrombosis.
PNH diagnosis was formerly comforted by in vitro complement activation by either acidity (Ham test) or osmolarity (sucrose test). These tests are obsolete as the diagnosis of PNH by flow cytometry (FCM) refers to the detection of the pathognomonic anomaly. GPI-anchored proteins can be detected after labeling the cells with monoclonal antibodies (for example, anti-CD55 or anti-CD59) or a reagent known as fluorescein-tagged proaerolysin (FLAER), which binds to the glycan portion of the GPI anchor. FLAER is best used on nucleated cells; it does not stain red blood cells, as red blood cells express high glycophorin levels, a protein that binds to aerolysin and, therefore, interferes with the assay.
Speaking of treatment, the only disease-modifying therapeutic strategies for PNH are complement inhibition therapy (eculizumab and ravulizumab) and bone marrow transplantation. Eculizumab and ravulizumab are the only licensed therapy for PNH, and their efficacy has relegated bone marrow transplantation to second-line therapy for hemolytic PNH in countries where the drug is available. Bone marrow transplantation might be an option if eculizumab is unavailable, and is a reasonable therapeutic strategy in patients with PNH and severe bone marrow failure. Adjunctive therapies (for example, immunosuppression) could be prescribed to patients with PNH who also have bone marrow failure to ameliorate the latter. However, these adjunctive treatments are not specific for PNH, nor do they have consistent effects on expanding or reducing PNH clones. The only curative strategy for PNH is allogeneic stem cell transplantation, but this procedure continues to carry a considerable risk of mortality.

Paroxysmal nocturnal hemoglobinuria Diagnosis and Treatment
It covers the details of conventional and current medical therapies and diagnosis available in the Paroxysmal nocturnal hemoglobinuria market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.
The Paroxysmal nocturnal hemoglobinuria market report gives a thorough understanding of Paroxysmal nocturnal hemoglobinuria by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides the treatment algorithms and treatment guidelines for Paroxysmal nocturnal hemoglobinuria in the US, Europe, and Japan.

Paroxysmal nocturnal hemoglobinuria Epidemiology
The Paroxysmal nocturnal hemoglobinuria epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven 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 report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings
The disease epidemiology covered in the report provides historical and forecasted Paroxysmal nocturnal hemoglobinuria epidemiology segmented as the Total diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria and Gender-specific Cases of PNH. The report includes the prevalent scenario of Paroxysmal nocturnal hemoglobinuria symptoms in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.

Country-wise Paroxysmal nocturnal hemoglobinuria Epidemiology
The epidemiology segment also provides the Paroxysmal nocturnal hemoglobinuria epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The total prevalent population of Paroxysmal nocturnal hemoglobinuria in the 7MM countries was estimated to be 8,385 cases in 2020.

Paroxysmal nocturnal hemoglobinuria Drug Chapters
The drug chapter segment of the Paroxysmal nocturnal hemoglobinuria report encloses the detailed analysis of Paroxysmal nocturnal hemoglobinuria marketed drugs and late stage (Phase-III, Phase-II/III, Phase-II, and Phase-I/II) pipeline drugs. It also helps to understand the Paroxysmal nocturnal hemoglobinuria 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.
Until 2007, PNH was a devastating disease with no treatment for hemolysis and thrombosis and the leading cause of deaths in PNH patients. However, with the advent of the anti-C5 agent-Eculizumab in the last decade, the treatment of PNH has been revolutionized. It not only reduced hemolysis and decreased the number of transfusions but most importantly reduced the rate of thrombosis. Ten years of experience with this drug provided evidence that mitigates initial concerns about infectious events, if the patient is well vaccinated the rate seems to be low and stable
Products detail in the report…
Paroxysmal nocturnal hemoglobinuria Emerging Drugs
Pegcetacoplan (APL-2) (Apellis Pharmaceuticals) is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, leading to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan has been granted Fast Track designation by the FDA for the treatment of PNH. Recently, ?Apellis submitted Marketing applications for pegcetacoplan to treat PNH to the FDA and the European Medicines Agency (EMA). Additionally, the FDA granted the application priority review designation and set a target action date of May 2021. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021.
Crovalimab (Hoffmann-La Roche) is also known as RG6107, and SKY59 is a humanized complement inhibitor C5 monoclonal antibody (anti-C5 recycling antibody) discovered by Chugai using recycling antibody technology. SKY59 is designed to target C5, a key component of the complement system, and is expected to control complement activity. It inhibits complement activation by blocking the cleavage of c5 to c5a and c5b, which is the cause of PNH. Roche is co-developing SKY59 with Chugai Pharmaceuticals. The drug has received orphan drug designation in the US and is currently being evaluated in phase III clinical studies in patients with PNH currently treated with complement inhibitors as well as not previously treated with complement inhibitors.
Products detail in the report…

Paroxysmal nocturnal hemoglobinuria Market Outlook
The Paroxysmal nocturnal hemoglobinuria market outlook of the report builds a detailed comprehension of the historical, current, and forecasted PNH market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand for better technology.
This segment gives a thorough detail of Paroxysmal nocturnal hemoglobinuria 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’s, 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.
According to the analyst, the Paroxysmal nocturnal hemoglobinuria market in the 7MM is expected to change in the study period 2017–2030.
Substantial progress in the biology and treatment of PNH has occurred over the past two decades, making PNH a model for progress in precision medicine. A thorough understanding of the molecular and cellular underpinnings of PNH has led to the development of a targeted therapy, which has altered the natural history of the disease. However, there is still room for improvement in the care of patients with PNH. The future approvals of PNH further move towards personalized patient-centered care with better options to reduce the frequency and self-administer medication. Auto-injections of SC compounds, as well as oral drugs, will increase patient autonomy, and compliance will become critical.

Key Findings
This section includes a glimpse of the Paroxysmal nocturnal hemoglobinuria market in the 7MM. The market size of Paroxysmal nocturnal hemoglobinuria in the seven major markets was found to be USD 358.04 million in 2020.

The United States: Market Outlook
This section provides the total Paroxysmal nocturnal hemoglobinuria market size and market size by therapies of Paroxysmal nocturnal hemoglobinuria in the United States.
The United States accounts for the highest market size of Paroxysmal nocturnal hemoglobinuria in comparison to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
Complement C5 inhibition is the standard of care (SoC) for patients with paroxysmal nocturnal hemoglobinuria (PNH) with significant clinical symptoms.
Alexion Pharmaceuticals-Soliris (Eculizumab) is the first generation of C5 inhibitor, binds to the complement protein C5, thus preventing terminal complement activation and subsequent intravascular hemolysis. It was licensed by the FDA and EMA in 2007. The product also received approval from PDMA and entered the Japan Market. Since then it has revolutionized the treatment of patients with the disease in countries with access to this therapy.
Although Soliris has provided a better quality of life to the patients by reducing the need for frequent blood transfusion higher dose requirement, painful mode of administration, the risk of meningococcal infection, and unable to eliminate the PNH clone and up to 20% of patients still experience symptomatic extravascular hemolysis that requires periodic blood transfusions. The major problems that need to be addressed include mitigating extravascular hemolysis in patients on eculizumab and treating the rare patients who are unresponsive to eculizumab owing to mutant C5. The development of ravulizumab, an eculizumab-like monoclonal antibody engineered to have a longer half-life, is intended to provide the same benefits as eculizumab with a more convenient and effective dosing schedule). It was approved by the FDA in December 2018.
Ravulizumab is expected to maintain Alexion Pharmaceuticals revenue and its position in the complement inhibitor space by converting the patient pool from the Soliris to the Ultomiris before the advent of biosimilars and other competitions in the PNH market.

EU-5 Countries: Market Outlook
The total Paroxysmal nocturnal hemoglobinuria market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are also mentioned.

Japan Market Outlook
The total Paroxysmal nocturnal hemoglobinuria market size and market size by therapies of Paroxysmal nocturnal hemoglobinuria in Japan are also mentioned.

Paroxysmal nocturnal hemoglobinuria Drugs Uptake
This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2017–2030. The analysis covers Paroxysmal nocturnal hemoglobinuria 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 allow 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.

Paroxysmal nocturnal hemoglobinuria Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stage. It also analyses Paroxysmal nocturnal hemoglobinuria key players involved in developing targeted therapeutics.
Major players include Apellis Pharmaceuticals, Hoffmann-La Roche, Novartis, Alexion Pharmaceutical, Regeneron Pharmaceuticals, RA Pharmaceuticals, and others, whose key products are expected to get launched in the US market by 20XX.

Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Paroxysmal nocturnal hemoglobinuria emerging therapies.
KOL Views
To keep up with current market trends, we take KOLs and SME’s opinion working in the Paroxysmal nocturnal hemoglobinuria 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 Paroxysmal nocturnal hemoglobinuria market trend. 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
We perform Competitive and Market Intelligence analysis of the Paroxysmal nocturnal hemoglobinuria market by using various Competitive Intelligence tools: 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.

Scope of the Report
• The report covers the descriptive overview of PNH, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
• Comprehensive insight has been provided into the Paroxysmal nocturnal hemoglobinuria epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Paroxysmal nocturnal hemoglobinuria is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
• A detailed review of the Paroxysmal nocturnal hemoglobinuria market; historical and forecasted, is included in the report, covering drug outreach in the 7MM
• The report provides an edge while developing business strategies by understanding trends shaping and driving the global Paroxysmal nocturnal hemoglobinuria market

Report Highlights
• In the coming years, the Paroxysmal nocturnal hemoglobinuria 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 Paroxysmal nocturnal hemoglobinuria R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
• Major players are involved in developing therapies for PNH. The launch of emerging therapies will significantly impact the PNH market
• A better understanding of disease pathogenesis will also contribute to developing novel therapeutics for PNH.

• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competitor, 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

Paroxysmal nocturnal hemoglobinuria Report Insights
• Patient Population
• Therapeutic Approaches
• Paroxysmal nocturnal hemoglobinuria Pipeline Analysis
• Paroxysmal nocturnal hemoglobinuria Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Paroxysmal nocturnal hemoglobinuria Report Key Strengths
• 10-years Forecast
• 7MM Coverage
• Paroxysmal nocturnal hemoglobinuria Epidemiology Segmentation
• Key Competitors
• Highly Analyzed Market

• Drugs Uptake

Paroxysmal nocturnal hemoglobinuria Report Assessment
• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers

Key Questions

Market Insights:
• What was the Paroxysmal nocturnal hemoglobinuria Market share (%) distribution in 2017, and how would it look like in 2030?
• What would be the Paroxysmal nocturnal hemoglobinuria total market Size and market Size by therapies across the 7MM during the forecast period (2017–2030)?
• What are the key findings pertaining to the market across 7MM, and which country will have the largest Paroxysmal nocturnal hemoglobinuria market Size during the forecast period (2017–2030)?
• At what CAGR, the Paroxysmal nocturnal hemoglobinuria market is expected to grow in the 7MM during the forecast period (2017–2030)?
• What would be the Paroxysmal nocturnal hemoglobinuria market outlook across the 7MM during the forecast period (2017–2030)?
• What would be the Paroxysmal nocturnal hemoglobinuria market growth till 2030, and what will be the resultant market Size in the year 2030?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:
• What are the disease risk, burden, and unmet needs of Paroxysmal nocturnal hemoglobinuria?
• What is the historical Paroxysmal nocturnal hemoglobinuria patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What would be the forecasted patient pool of Paroxysmal nocturnal hemoglobinuria in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Paroxysmal nocturnal hemoglobinuria?
• Out of all 7MM countries, which country would have the highest prevalence of Paroxysmal nocturnal hemoglobinuria during the forecast period (2017–2030)?
• At what CAGR the population is expected to grow in the 7MM during the forecast period (2017–2030)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:
• What are the current options for the treatment of Paroxysmal nocturnal hemoglobinuria?
• What are the current treatment guidelines for treating Paroxysmal nocturnal hemoglobinuria in the USA, Europe, and Japan?
• What are the Paroxysmal nocturnal hemoglobinuria marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of Paroxysmal nocturnal hemoglobinuria?
• How many therapies are developed by each company for the treatment of Paroxysmal nocturnal hemoglobinuria?
• How many emerging therapies are in the mid-stage and late stages of development for the treatment of Paroxysmal nocturnal hemoglobinuria?
• What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Paroxysmal nocturnal hemoglobinuria therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Paroxysmal nocturnal hemoglobinuria and their status?
• What are the key designations that have been granted for the emerging therapies for Paroxysmal nocturnal hemoglobinuria?
• What are the global historical and forecasted market for Paroxysmal nocturnal hemoglobinuria?

Reasons to buy
• The report will help in developing business strategies by understanding trends shaping and driving the Paroxysmal nocturnal hemoglobinuria market
• To understand the future market competition in the Paroxysmal nocturnal hemoglobinuria market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Paroxysmal nocturnal hemoglobinuria in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the Paroxysmal nocturnal hemoglobinuria market.
• To understand the future market competition in the Paroxysmal nocturnal hemoglobinuria market.