Paroxysmal Nocturnal Hemoglobinuria Market Landscape Set to Witness Growth at a CAGR of 10% for the Study Period 2018-30 in the 7MM | DelveInsight

LAS VEGAS, March 23, 2021 /PRNewswire/ -- DelveInsight's 'Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Insights, Epidemiology, and Market Forecast' report offers detailed coverage of historical and forecasted epidemiological analysis in the 7MM (the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan) for the study period 2018-30. 

The report is created to project true opportunities in the market landscape, help clients form collaborations and agreements that give them a competitive advantage, and plan strategic moves to yield maximum ROI. 

Some of the crucial highlights from the Paroxysmal Nocturnal Hemoglobinuria Market report: 

• The total PNH prevalent cases in the 7MM were estimated to be 8,385 in 2020 and is expected to increase owing to various factors during the forecasted period.
• The only disease-modifying therapies in the PNH therapy market are complement inhibition therapy (Alexion Pharmaceuticals' Eculizumab and Ravulizumab) and bone marrow transplantation. 
• Eculizumab has managed to reduce hemolysis, decrease the number of the required number of transfusions, as well as reducing the rate of thrombosis.
• Alexion developed another eculizumab-like monoclonal antibody, Ravulizumab, however, is more convenient to administer and has an effective dosing schedule than Eculizumab, thus, is expected to add to the company's revenue. . 
• The present PNH emerging market consists of second-generation C5 Inhibitors, C3 inhibitors, factor D and Factor B inhibitors. 
• Key pharmaceutical companies operating in the Paroxysmal Nocturnal Hemoglobinuria therapeutics market are Apellis Pharmaceuticals, Hoffmann-La Roche, Novartis, Alexion Pharmaceutical, Regeneron Pharmaceuticals, RA Pharmaceuticals and others. 
• The total PNH market size, as per DelveInsght's market analysis, was found to be USD 358.04 million in 7MM in 2020. In the 7MM, the US accounted for approximately 63% of the overall market size of PNH in 2020. 
• A number of emerging therapies comprising mAb–derived compounds, siRNA, C3 inhibitors, Factor B and D inhibitors are currently in the PNH pipeline.
• The Paroxysmal Nocturnal Hemoglobinuria market has several unmet needs such as hemolysis, anemia, transfusions, and severe fatigue that might affect bone marrow function, which need to be addressed. 
• Further, the launch of self-administered therapies in the Paroxysmal Nocturnal Hemoglobinuria therapy market is expected to lower down the treatment burden. 

Want to know more? Get comprehensive insights @ Paroxysmal Nocturnal Hemoglobinuria Market Landscape, Epidemiological Insights, and Pipeline Therapies 

The Paroxysmal Nocturnal Hemoglobinuria market report offers available therapies, treatment landscape, emerging therapies, changing market landscape, individual market share of pipeline therapies, PNH market drivers, and market barriers, PNH market size during the study period 2018-30 in the 7MM (the US, EU5 (the UK, Germany, Italy, Spain and France), and Japan).

Paroxysmal Nocturnal Hemoglobinuria is a rare acquired disorder of the pluripotent hematopoietic stem cell; therefore, it can affect erythrocytes, leukocytes, thrombocytes, and probably some endothelial cells. Paroxysmal Nocturnal Hemoglobinuria diagnosis is made based on clinical evaluation, patient history, and several tests that are specialized to identify Paroxysmal Nocturnal Hemoglobinuria cells such as flow cytometry. 

Paroxysmal Nocturnal Hemoglobinuria symptoms often include fatigue, difficulty in swallowing (dysphagia), shortness of breath (dyspnea), pain in the abdomen, anemia, erectile dysfunction, and dark-colored urine (hemoglobinuria). However, hemolysis in Paroxysmal Nocturnal Hemoglobinuria that leads to thrombosis remains to be the most devastating consequence of the blood disorder and can often prove to be fatal. 

 Paroxysmal Nocturnal Hemoglobinuria Epidemiological Analysis & Forecast

Paroxysmal Nocturnal Hemoglobinuria is an ultra-rare blood disorder, and DelveInsight's 7MM Paroxysmal Nocturnal Hemoglobinuria epidemiological analysis demonstrates that it is primarily more common in females than males. Further, the US accounted for the highest diagnosed prevalent cases, approximately 51% of the total cases in the 7MM, followed by Germany. 

The PNH market analysis report proffers historical as well as forecasted epidemiological segmentation for the study period 2018-30: 

• Total diagnosed Paroxysmal Nocturnal Hemoglobinuria prevalent cases 
• Gender-specific Paroxysmal Nocturnal Hemoglobinuria cases 

Dive deeper for rich insights into Paroxysmal Nocturnal Hemoglobinuria Epidemiological Analysis 

Paroxysmal Nocturnal Hemoglobinuria Treatment Landscape 

Before the approval of anti-C5 agent-Eculizumab, Paroxysmal Nocturnal Hemoglobinuria was a devastating disease that would lead to breaking of red blood cells prematurely. However, Eculizumab transformed the Paroxysmal Nocturnal Hemoglobinuria treatment landscape. The therapeutic agent not only helped in the management of hemolysis, but also helped in reducing the rate of thrombosis and the number of transfusions. It managed to push bone marrow transplantation to second-line therapy for hemolytic PNH in countries where the drug is available. 

Visit to know more about Paroxysmal Nocturnal Hemoglobinuria Marketed Drugs 

Paroxysmal Nocturnal Hemoglobinuria Market 

The Paroxysmal Nocturnal Hemoglobinuria market is experiencing an influx of several pharmaceutical companies investigating numerous second-generation C5 inhibitors to bridge the treatment gap. Key pharmaceutical players such as Apellis Pharmaceuticals, Hoffmann-La Roche, Novartis, Alexion Pharmaceutical, Regeneron Pharmaceuticals, RA Pharmaceuticals and several others are energetically operating in the domain. 

The PNH pipeline therapies involve second-generation C5 Inhibitors, C3 inhibitors, factor D and Factor B inhibitors expected to get launched in the coming decade and revolutionize the Paroxysmal Nocturnal Hemoglobinuria market owing to the option of self-administration that shall significantly lower down the treatment burden. However, there is a need for clinical trials that study the efficiency of emerging therapies in multicentric versus reference laboratory settings. 

There are several unmet needs in the PNH market in the form of the availability of limited treatment options, high rates of morbidity in PNH patients, and a lack of well-defined protocols to manage the disorder. Moreover, the rarity of the disorder also causes unawareness among the physicians and people regarding Paroxysmal Nocturnal Hemoglobinuria symptoms, treatment and diagnosis. 

Interested in knowing more? Drop by @ Paroxysmal Nocturnal Hemoglobinuria Market Domain and Key Companies

Nevertheless, the studies demonstrated that terminal complement blockade offers a safe and effective treatment option to PNH patients. The Paroxysmal Nocturnal Hemoglobinuria drug pipeline involves several alternative therapies with convenient RoA that have increased half-life which reduces the dose-burden and improved the quality of life. Better diagnosis modalities for such rare diseases are as important as their treatments. Better diagnostics methods over the past decade undoubtedly have lowered down the PNH treatment burden. 

 Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapies 

• Pegcetacoplan (APL-2): Apellis Pharmaceuticals
• Crovalimab: Hoffmann-La Roche 
• Iptacopan or LNP023: Novartis
• ALXN2040 (or Danicopan): Alexion Pharmaceutical
• Pozelimab: Regeneron Pharmaceuticals
• RA101495: UCB Pharma/RA Pharmaceuticals

Know more about the pipeline therapies @ PNH Emerging Drug Therapies 

Scope of the Report 

Coverage: 7MM (the US, EU5, and Japan)

Study Period: 2018-30

Key Companies: Apellis Pharmaceuticals, Hoffmann-La Roche, Novartis, Alexion Pharmaceutical, Regeneron Pharmaceuticals, RA Pharmaceuticals and several others.

Key Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapies: Pegcetacoplan (APL-2), Crovalimab, Iptacopan or LNP023, ALXN2040 (or Danicopan), Pozelimab, RA101495, and others. 

Paroxysmal Nocturnal Hemoglobinuria Market Segmentation: By Geography, By Paroxysmal Nocturnal Hemoglobinuria Therapies

Analysis: Comparative and conjoint analysis of Paroxysmal Nocturnal Hemoglobinuria emerging therapies

Tools used: SWOT analysis, Conjoint Analysis, Porter's Five Forces, PESTLE analysis, BCG Matrix analysis methods.

Case Studies

KOL's Views

Analyst's Views

Get in touch to know more about the report scope @ PNH Market Landscape and Future Trends 

Table of Contents

Know more about the report offerings @ Paroxysmal Nocturnal Hemoglobinuria Market 

Related Reports 

Hemophilia B Market

Get comprehensive historical and forecast analysis of Hemophilia B Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as UniQure Biopharma B.V./ CSL Behring, Pfizer/Spark Therapeutics, Genzyme (Sanofi Company/ Alnylam Pharmaceuticals), Novo Nordisk, UniQure Biopharma B.V., ApcinteX, Freeline Therapeutics, Sangamo Therapeutics and several others. 

Wilson Disease Market

Get comprehensive historical and forecast analysis of Wilson Disease Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Vivet Therapeutics, Cuprior, GMP-Orphan SAS and Alexion, and several others. 

Eosinophilic Esophagitis Market

Get comprehensive historical and forecast analysis of Eosinophilic Esophagitis Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Shire, Dr. Falk GmBH, Adare Pharmaceuticals, and Regeneron.

Von Willebrand Disease Market

Get comprehensive historical and forecast analysis of Von Willebrand Disease Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Shire Plc. (earlier Baxalta), CSL Behring, LFB Biomedicaments SA, Octapharma AG, Grifols SA, and others. 

Familial Chylomicronemia Syndrome Market

Get comprehensive historical and forecast analysis of Familial Chylomicronemia Syndrome Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Akcea Therapeutics/Ionis Pharmaceuticals, Arrowhead Pharmaceuticals, Amryt Pharma, and others. 

Adrenoleukodystrophy Market

Get comprehensive historical and forecast analysis of Adrenoleukodystrophy Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Bluebird Bio, Minoryx Therapeutics, Magenta Therapeutics, and others. 

Lamellar Ichthyosis Market

Get comprehensive historical and forecast analysis of the Lamellar Ichthyosis Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Galderma, Mayne, Pharma, Krystal Biotech, Timber Pharmaceuticals, and others. 

Pulmonary Arterial Hypertension Market

Get comprehensive historical and forecast analysis of the Lamellar Ichthyosis Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as United Therapeutics, Arena Pharmaceuticals, Altavant Sciences, Acceleron Pharma, Actelion Pharmaceuticals, and others. 

Duchenne Muscular Dystrophy Market

Get comprehensive historical and forecast analysis of the Duchenne Muscular Dystrophy Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Sarepta Therapeutics, FibroGen, Capricor, Santhera Pharmaceutical, Catabasis Pharmaceuticals, Italfarmaco, and others. 

Warm Autoimmune Hemolytic Anemia Market

Get comprehensive historical and forecast analysis of the Duchenne Muscular Dystrophy Market, Epidemiological and Therapeutics market domain with a special focus on key companies such as Rigel Pharmaceuticals, Apellis Pharmaceuticals, Momenta Pharmaceuticals, Alexion, Immunovant, and others.

Get in touch with our Business Executive @ Rare disease market insights to know more. 

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance.  Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

Contact Us

Shruti Thakur 
[email protected] 
+1(919)321-6187 
www.delveinsight.com

SOURCE DelveInsight Business Research, LLP