Established SoC, first C5 inhibitors are set to be challenged in the Paroxysmal Nocturnal Hemoglobinuria (PNH) market by second generation C5 inhibitors and novel C3 inhibitors.
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.
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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
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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.
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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.
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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
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Table of Contents
1 | Key Insights |
2 | Report Introduction |
3 | Competitive Intelligence Analysis for Paroxysmal Nocturnal Hemoglobinuria |
4 | Paroxysmal Nocturnal Hemoglobinuria Market Overview at a Glance |
5 | Executive Summary of Paroxysmal Nocturnal Hemoglobinuria |
6 | PNH Epidemiology and Market Methodology |
7 | Paroxysmal Nocturnal Hemoglobinuria Epidemiology and Patient Population |
8 | PNH Patient Journey |
9 | Treatment Algorithm, Current Treatment, and Medical Practices |
10 | Key Endpoints in PNH Clinical Trials |
11 | PNH Marketed Therapies |
12 | Paroxysmal Nocturnal Hemoglobinuria Emerging Therapies |
13 | Paroxysmal Nocturnal Hemoglobinuria: 7 Major Market Analysis |
14 | Attribute analysis |
15 | Access and Reimbursement Overview of Paroxysmal Nocturnal Hemoglobinuria |
16 | KOL Reviews |
17 | Case Reports |
18 | Paroxysmal Nocturnal Hemoglobinuria Market Drivers |
19 | PNH Market Barriers |
20 | SWOT Analysis |
21 | Disclaimer |
21 | DelveInsight Capabilities |
22 | About DelveInsight |
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