Paroxysmal Nocturnal Hemoglobinuria Treatment Market 2034: Clinical Trials, Medication, Pipeline Therapies, EMA, PDMA, FDA Approvals and Companies by DelveInsight

Paroxysmal Nocturnal Hemoglobinuria Market

(Albany, USA) DelveInsight’s “Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology and Market Forecast– 2034” by DelveInsight provides a comprehensive analysis of Paroxysmal Nocturnal Hemoglobinuria, including a detailed examination of its historical and projected Paroxysmal Nocturnal Hemoglobinuria epidemiology. Additionally, the report offers insights into the Paroxysmal Nocturnal Hemoglobinuria market trends in the United States, EU-5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan, aiming to enhance the understanding of this condition. 

The Paroxysmal Nocturnal Hemoglobinuria market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Paroxysmal Nocturnal Hemoglobinuria market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Paroxysmal Nocturnal Hemoglobinuria treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Paroxysmal Nocturnal Hemoglobinuria market.

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Some facts of the Paroxysmal Nocturnal Hemoglobinuria Market Report are:

• According to DelveInsight, Paroxysmal Nocturnal Hemoglobinuria market size in the 7MM is expected to reach USD ~2,500.4 million by 2034.
• Leading Paroxysmal Nocturnal Hemoglobinuria companies working in the treatment market are Hoffmann-La Roche, Alexion Pharmaceuticals, Novartis, Regeneron Pharmaceuticals, BioCryst Pharmaceuticals, Apellis Pharmaceuticals, Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio, and others.
• Promising Paroxysmal Nocturnal Hemoglobinuria Therapies expected to launch in the market are Crovalimab, Danicopan, Iptacopan, Pozelimab (REGN3918) + Cemdisiran, BCX9930, and others.
• DelveInsight estimates that there were approximately 12K prevalent cases of paroxysmal nocturnal hemoglobinuria in the 7MM in 2021.
• On April 2023, BioCryst Pharmaceuticals announced a study which is designed to provide continued access to BCX9930 for subjects currently receiving treatment with BCX9930 in a BioCryst-sponsored clinical study for paroxysmal nocturnal hemoglobinuria (PNH) who, in the opinion of the investigator, would benefit from continued treatment with BCX9930 and who do not have access to other treatment options.
• On June 2023, Novartis announced a study on iptacopan. This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan

Paroxysmal Nocturnal Hemoglobinuria Overview

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological disorder characterized by intravascular hemolysis, hemoglobinuria, hemolysis, and venous thrombosis. Somatic PIGA mutations cause PNH in one or more HSC clones. The PIGA gene product is required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIGA mutations result in a deficiency of GPI-anchored proteins, such as complement decay-accelerating factor (CD55) and CD59 glycoprotein (CD59), both of which are complement inhibitors.

PNH is also known as the “most vicious acquired thrombophilic state,” most likely due to free hemoglobin and decreased intravascular nitric oxide, though its multifactorial biological basis is unknown. A clinical evaluation, patient history, and other factors are used to make a paroxysmal nocturnal hemoglobinuria diagnosis. Flow cytometry is the primary diagnostic test for people who have PNH. Urine hemosiderin is almost always present in PNH and can be used to screen for the disorder.

Learn more about Paroxysmal Nocturnal Hemoglobinuria treatment algorithms in different geographies, and patient journeys. Contact to receive a sample at: https://www.delveinsight.com/sample-request/paroxysmal-nocturnal-hemoglobinuria-market

Paroxysmal Nocturnal Hemoglobinuria Market 

The Paroxysmal Nocturnal Hemoglobinuria market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Paroxysmal Nocturnal Hemoglobinuria market trends by analyzing the impact of current Paroxysmal Nocturnal Hemoglobinuria therapies on the market and unmet needs, and drivers, barriers, and demand for better technology.

This segment gives a thorough detail of the 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, 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 Paroxysmal Nocturnal Hemoglobinuria market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Paroxysmal Nocturnal Hemoglobinuria market in 7MM is expected to witness a major change in the study period 2020-2034.

Paroxysmal Nocturnal Hemoglobinuria Epidemiology 

The Paroxysmal Nocturnal Hemoglobinuria epidemiology section provides insights into the historical and current Paroxysmal Nocturnal Hemoglobinuria patient pool and forecasted trends for seven individual 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 Paroxysmal Nocturnal Hemoglobinuria market report also provides the diagnosed patient pool, trends, and assumptions. 

Explore more about Paroxysmal Nocturnal Hemoglobinuria Epidemiology @ Paroxysmal Nocturnal Hemoglobinuria Prevalence

Paroxysmal Nocturnal Hemoglobinuria Drugs Uptake

This section focuses on the uptake rate of the potential Paroxysmal Nocturnal Hemoglobinuria drugs recently launched in the Paroxysmal Nocturnal Hemoglobinuria market or expected to be launched in 2020-2034. The analysis covers the Paroxysmal Nocturnal Hemoglobinuria market uptake by drugs, patient uptake by therapies, and sales of each drug.   

Paroxysmal Nocturnal Hemoglobinuria Drugs Uptake helps in understanding the 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 Paroxysmal Nocturnal Hemoglobinuria market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Paroxysmal Nocturnal Hemoglobinuria Pipeline Development Activities

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

• Crovalimab: Hoffmann-La Roche
• Danicopan: Alexion Pharmaceuticals
• Iptacopan: Novartis
• Pozelimab (REGN3918) + Cemdisiran: Regeneron Pharmaceuticals
• BCX9930: BioCryst Pharmaceuticals

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Scope of the Paroxysmal Nocturnal Hemoglobinuria Market Report:

• Study Period: 2020–2034
• Coverage: 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]
• Paroxysmal Nocturnal Hemoglobinuria Market Size in 2023: USD ~1400 million
• Key Paroxysmal Nocturnal Hemoglobinuria Companies: Hoffmann-La Roche, Alexion Pharmaceuticals, Novartis, Regeneron Pharmaceuticals, BioCryst Pharmaceuticals, Apellis Pharmaceuticals, Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, RallyBio, and others
• Key Pipeline Paroxysmal Nocturnal Hemoglobinuria Therapies: Crovalimab, Danicopan, Iptacopan, Pozelimab (REGN3918) + Cemdisiran, BCX9930, and others

Learn more about the emerging Paroxysmal Nocturnal Hemoglobinuria therapies & key companies @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials and Medication

Paroxysmal Nocturnal Hemoglobinuria Report Key Insights

1. Paroxysmal Nocturnal Hemoglobinuria Patient Population

2. Paroxysmal Nocturnal Hemoglobinuria Market Size and Trends

3. Key Cross Competition in the Paroxysmal Nocturnal Hemoglobinuria Market

4. Paroxysmal Nocturnal Hemoglobinuria Market Dynamics (Key Drivers and Barriers)

5. Paroxysmal Nocturnal Hemoglobinuria Market Opportunities

6. Paroxysmal Nocturnal Hemoglobinuria Therapeutic Approaches

7. Paroxysmal Nocturnal Hemoglobinuria Pipeline Analysis

8. Paroxysmal Nocturnal Hemoglobinuria Current Treatment Practices/Algorithm

9. Impact of Emerging Therapies on the Paroxysmal Nocturnal Hemoglobinuria Market

Table of Contents

1. Key Insights

2. Executive Summary

3. Paroxysmal Nocturnal Hemoglobinuria Competitive Intelligence Analysis

4. Paroxysmal Nocturnal Hemoglobinuria Market Overview at a Glance

5. Paroxysmal Nocturnal Hemoglobinuria Disease Background and Overview

6. Paroxysmal Nocturnal Hemoglobinuria Patient Journey

7. Paroxysmal Nocturnal Hemoglobinuria Epidemiology and Patient Population

8. Paroxysmal Nocturnal Hemoglobinuria Treatment Algorithm, Current Treatment, and Medical Practices

9. Paroxysmal Nocturnal Hemoglobinuria Unmet Needs

10. Key Endpoints of Paroxysmal Nocturnal Hemoglobinuria Treatment

11. Paroxysmal Nocturnal Hemoglobinuria Marketed Products

12. Paroxysmal Nocturnal Hemoglobinuria Emerging Therapies

13. Paroxysmal Nocturnal Hemoglobinuria Seven Major Market Analysis

14. Attribute Analysis

15. Paroxysmal Nocturnal Hemoglobinuria Market Outlook (7 major markets)

16. Paroxysmal Nocturnal Hemoglobinuria Access and Reimbursement Overview

17. KOL Views on the Paroxysmal Nocturnal Hemoglobinuria Market

18. Paroxysmal Nocturnal Hemoglobinuria Market Drivers

19. Paroxysmal Nocturnal Hemoglobinuria Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

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