Paroxysmal Nocturnal Hemoglobinuria Market to Climb Moderately at a CAGR of 7.6% During the Study Period (2019-2032), Assesses DelveInsight

Rhea-AI Summary

The paroxysmal nocturnal hemoglobinuria (PNH) market is projected to reach USD 1,100 million in 2021, with anticipated growth driven by rising cases and new therapies from major companies like Hoffmann-La Roche, Alexion, and Novartis. DelveInsight's report indicates approximately 12K prevalent cases in the 7MM (U.S., EU-4, U.K., Japan) as of 2021. Key pipeline therapies include Crovalimab and Iptacopan. Despite advancements, challenges like continuous hemolysis and the impending expiration of eculizumab patents in 2024 and 2027 could impact market dynamics. This report covers market trends through 2032, focusing on treatment efficacy, barriers, and emerging drugs.

Positive

• Projected market size of USD 1,100 million in 2021.
• Estimated 12K prevalent cases in the 7MM.
• Anticipated growth driven by new emerging therapies.
• Development of novel drugs by leading companies.

Negative

• Potential market impact from eculizumab patent expiration in 2024/2027.
• Patients may continue to experience hemolysis and other complications.
• Limited physician awareness of PNH's symptoms affecting diagnosis.

The paroxysmal nocturnal hemoglobinuria market is expected to show positive growth, mainly attributed to the increasing cases and also, the launch of upcoming therapies during the forecast period. Moreover, advances in disease mechanisms have yielded new diagnostic and therapeutic approaches, opening the way to more drug development

LAS VEGAS, Jan. 23, 2023 /PRNewswire/ -- DelveInsight's Paroxysmal Nocturnal Hemoglobinuria Market Insights report includes a comprehensive understanding of current treatment practices, paroxysmal nocturnal hemoglobinuria emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Takeaways from the Paroxysmal Nocturnal Hemoglobinuria Market Report

• As per DelveInsight analysis, the paroxysmal nocturnal hemoglobinuria market size in the 7MM was approximately USD 1,100 million in 2021.
• According to the assessment done by DelveInsight, the estimated total paroxysmal nocturnal hemoglobinuria prevalent cases in the 7MM were approximately 12K in 2021. 
• Globally, leading paroxysmal nocturnal hemoglobinuria companies such as 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 are developing novel paroxysmal nocturnal hemoglobinuria drugs that can be available in the paroxysmal nocturnal hemoglobinuria market in the coming years.
• The promising paroxysmal nocturnal hemoglobinuria therapies in the pipeline include Crovalimab, Danicopan, Iptacopan, Pozelimab (REGN3918) + Cemdisiran, BCX9930, and others.
• In December 2022, Novartis released the efficacy and adverse event data from the phase III APPLY-PNH trial in paroxysmal nocturnal hemoglobinuria.
• In September 2022, efficacy and safety data from a phase III trial in paroxysmal nocturnal hemoglobinuria were released by Alexion.
• In August 2022, BioCryst announced that the US FDA had lifted its partial clinical hold on the BCX9930 program. The company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930.
• In May, Regeneron Pharmaceuticals completed a phase III trial in Paroxysmal nocturnal hemoglobinuria in the Czech Republic, Hungary, Netherlands, Italy, South Korea, and the United Kingdom.
• In April 2022, BioCryst voluntarily paused enrollment in BCX9930 clinical trials while investigating elevated serum creatinine (SCr) in some patients at the 500 mg twice daily dose. The FDA subsequently placed the program on a partial clinical hold. 

Discover which therapies are expected to grab the major paroxysmal nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report

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.

Paroxysmal Nocturnal Hemoglobinuria Epidemiology Segmentation

DelveInsight estimates that there were approximately 12K prevalent cases of paroxysmal nocturnal hemoglobinuria in the 7MM in 2021.

Among the EU4 countries, Germany had the highest number of cases of PNH in 2021, followed by France.

The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:

• Total Diagnosed Prevalence
• Gender-specific Prevalence

Download the report to understand which factors are driving paroxysmal nocturnal hemoglobinuria epidemiology trends @ Paroxysmal Nocturnal Hemoglobinuria Epidemiological Insights

Paroxysmal Nocturnal Hemoglobinuria Treatment Market 

Complement inhibition therapy and bone marrow transplantation are two disease-modifying therapeutic strategies for PNH. Eculizumab is a licensed therapy for PNH, and its efficacy has relegated bone marrow transplantation to a secondary treatment option for hemolytic PNH in countries where the drug is available. If eculizumab is unavailable, bone marrow transplantation is a viable therapeutic option for patients with PNH and severe bone marrow failure. Adjunctive therapies (such as immunosuppression) could be used to treat PNH patients with bone marrow failure. These complementary treatments, however, are not specific to PNH and do not have consistent effects on the expansion or reduction of PNH clones. Allogeneic stem cell transplantation is the only curative strategy for PNH, but it still carries a significant mortality risk and is only considered for patients with severe bone marrow failure.

PNH was a devastating disease with no treatment for hemolysis and thrombosis until 2007, and it was the leading cause of death in PNH patients. However, since the introduction of the anti-C5 agent eculizumab in the last decade, the paroxysmal nocturnal hemoglobinuria treatment has been transformed. It reduced hemolysis, the number of transfusions, and, most importantly, the thrombosis rate. Ten years of experience with this drug has provided evidence to alleviate initial concerns about infectious events; if the patient is adequately vaccinated, the rate appears to be low and stable.

To know more about paroxysmal nocturnal hemoglobinuria treatment guidelines, visit @ Paroxysmal Nocturnal Hemoglobinuria Management 

Paroxysmal Nocturnal Hemoglobinuria Pipeline Therapies and Key Companies

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

Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment 

Paroxysmal Nocturnal Hemoglobinuria Market Dynamics

The paroxysmal nocturnal hemoglobinuria market is anticipated to grow positively, mainly attributed to the increasing cases and also, the launch of upcoming therapies during the forecast period. In addition, over time, a better understanding of the role of uncontrolled complement activation in PNH has significantly improved the diagnosis and management of the disease. Moreover, agents such as mAb-derived compounds, C3 inhibitors, factor B, and Dinhibitors are currently in development to treat PNH and are expected to be available in the paroxysmal nocturnal hemoglobinuria market in the coming years.

Furthermore, in the last ten years, the C5 targeting monoclonal antibody eculizumab has changed the natural history of PNH. This is a fantastic opportunity to create new biomarkers for assessing thrombotic risk in PNH patients. Additionally, companies may be able to charge premium prices for their drugs if their therapies outperform current therapies, which thus boosts the paroxysmal nocturnal hemoglobinuria market growth.

However, despite C5 inhibitor treatment, patients may still experience continuous hemolysis, persistent chronic anemia, the need for ongoing blood transfusions, fatigue, and poor health-related quality of life. Physicians are generally unaware of the disease's broad spectrum of signs and symptoms, which are frequently similar to those of other diseases and vary from patient to patient.

Moreover, as the patent on eculizumab expires in 2027 in the United States and 2024 in the European Union, the introduction of biosimilars will result in the introduction of cheaper products to the paroxysmal nocturnal hemoglobinuria market, affecting the overall paroxysmal nocturnal hemoglobinuria market size. Furthermore, only a few studies have been conducted to compare the efficacy of medical indications such as PNH in multicentric vs. reference laboratory settings. Hence, all these factors will likely to hamper the paroxysmal nocturnal hemoglobinuria market growth.

Report Metrics	Details
Study Period	2019–2032
Coverage	7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]
Base Year	2019
Paroxysmal Nocturnal Hemoglobinuria Market CAGR	7.6 %
Paroxysmal Nocturnal Hemoglobinuria Market Size in 2021	USD 1,100 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

 

Scope of the Paroxysmal Nocturnal Hemoglobinuria Market Report

• Therapeutic Assessment: Paroxysmal Nocturnal Hemoglobinuria current marketed and emerging therapies
• Paroxysmal Nocturnal Hemoglobinuria Market Dynamics: Paroxysmal Nocturnal Hemoglobinuria market drivers and barriers 
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Unmet Needs, KOL's views, Analyst's views, Paroxysmal Nocturnal Hemoglobinuria Market Access and Reimbursement

Discover more about paroxysmal nocturnal hemoglobinuria drugs in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials

Table of Contents

1.	Paroxysmal Nocturnal Hemoglobinuria Market Key Insights
2.	Paroxysmal Nocturnal Hemoglobinuria Market Report Introduction
3.	Paroxysmal Nocturnal Hemoglobinuria Market Overview at a Glance
4.	Paroxysmal Nocturnal Hemoglobinuria Market Executive Summary
5.	Disease Background and Overview
6.	Paroxysmal Nocturnal Hemoglobinuria Treatment and Management
7.	Paroxysmal Nocturnal Hemoglobinuria Epidemiology and Patient Population
8.	Patient Journey
9.	Paroxysmal Nocturnal Hemoglobinuria Marketed Drugs
10.	Paroxysmal Nocturnal Hemoglobinuria Emerging Drugs
11.	Seven Major Paroxysmal Nocturnal Hemoglobinuria Market Analysis
12.	Paroxysmal Nocturnal Hemoglobinuria Market Outlook
13.	Potential of Current and Emerging Therapies
14.	KOL Views
15.	Paroxysmal Nocturnal Hemoglobinuria Market Drivers
16.	Paroxysmal Nocturnal Hemoglobinuria Market Barriers
17.	Unmet Needs
18.	SWOT Analysis
19.	Appendix
20.	DelveInsight Capabilities
21.	Disclaimer
22.	About DelveInsight

 

Related Reports

Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast

Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted paroxysmal nocturnal hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Paroxysmal Nocturnal Hemoglobinuria Pipeline

Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others.

Aplastic Anemia Market

Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others.

Aplastic Anemia Pipeline

Aplastic Anemia Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others.

Iron Deficiency Anemia Market

Iron Deficiency Anemia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key iron deficiency anemia companies, including Akebia Therapeutics, Shield Therapeutics, AMAG Pharmaceuticals, Pharmacosmos Therapeutics, Vifor Pharma, among others.

Iron Deficiency Anemia Pipeline

Iron Deficiency Anemia Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key iron deficiency anemia companies, including Akebia Therapeutics, Shield Therapeutics, AMAG Pharmaceuticals, Pharmacosmos Therapeutics, Vifor Pharma, among others.

Other Trending Reports

Tay-sachs Disease Or Gm2 Gangliosidosis Market | Onycholysis Market | Diagnostic Imaging Equipment Market | Chemotherapy-induced Peripheral Neuropathy Market | Global Electrophysiology Devices Market | Anaphylaxis Market | Atherectomy Devices Market | Helicobacter Pylori Infections Market | Ophthalmic Imaging Equipment Market | Androgenetic Alopecia Market | Allergic Rhinitis Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Chronic Inflammtory Demyelinating Polyneuropathy Market | Colorectal Cancer Crc Market | Opioid Induced Constipation Market | Vertigo Market | Bone Anchored Hearing Systems Market | Wound Closure Devices Market | Hip Replacement Devices Market | Hemodynamic Monitoring Systems Market | Egfr Non-small Cell Lung Cancer Market | Helicobacter Pylori Infection Market | Hyperkalemia Market | Polycythemia Market Neurostimulation Devices Market | Carpal Tunnel Syndrome Market | Ventilator Market | Cerebral Aneurysm Market | Alpha Antitrypsin Market | Binge Eating Disorder Market | Bunion Market | Concussions Market Size | Exocrine Pancreatic Insufficiency Market | Healthcare Due Diligence Services | Minimal Residual Disease Market | Hypertrophic Scar Market | Lung Fibrosis Market | Anterior Uveitis Market | 22q11.2 deletion syndrome Market | X-Linked Retinitis Pigmentosa (XLRP) Market | Acute Radiation Syndrome Market | Alpha-1 Protease Inhibitor Deficiency Market | Androgenetic Alopecia Market | Hyperlipidemia Market | Cardiotoxicity Market | Hypertrophic Cardiomyopathy Market | Fatty Acid Oxidation Disorders (FAODs) Market | Androgen Insensitivity Syndrome Market | Emphysema Market | Canaloplasty Market | Dravet Syndrome Market | Celiac Disease Market | Chlamydia Infections Market | Syphilis Market | Renal Tubular Acidosis Market | Palmoplantar Pustulosis (PPP) Market | Aplastic Anemia Market | Bacterial Pneumonia Market | B cell Chronic Lymphocytic Leukemia Market | B cell Lymphomas Market | Behcets Disease Market Neoantigen-based Personalized Cancer therapeutic Vaccines Competitive Landscape and Market Forecast—by 2035 | Glioblastoma Market

Related Healthcare Services

Healthcare Business Consulting

Healthcare Competitive Intelligence Services

Healthcare Asset Prioritization Services

About DelveInsight

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

Connect with us on LinkedIn|Facebook|Twitter

Additionally, get in touch with our business executive to explore @Healthcare Due Diligence Services 

Contact Us
Shruti Thakur 
info@delveinsight.com
+1(919)321-6187 
www.delveinsight.com

Logo: https://mma.prnewswire.com/media/1082265/DelveInsight_Logo.jpg

View original content:https://www.prnewswire.com/news-releases/paroxysmal-nocturnal-hemoglobinuria-market-to-climb-moderately-at-a-cagr-of-7-6-during-the-study-period-20192032-assesses-delveinsight-301727841.html

SOURCE DelveInsight Business Research, LLP

FAQ

What is the projected growth of the PNH market by 2032?

The PNH market is expected to grow significantly due to increasing cases and new therapies.

How many prevalent cases of PNH were reported in 2021?

Approximately 12K prevalent cases of PNH were reported in the 7MM in 2021.

Who are the major companies involved in PNH drug development?

Major companies include Hoffmann-La Roche, Alexion Pharmaceuticals, and Novartis.

What are some key pipeline therapies for PNH?

Key pipeline therapies include Crovalimab, Iptacopan, and Pozelimab.

What challenges is the PNH market facing?

Challenges include potential market impact from eculizumab's patent expiration and ongoing patient health issues.