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

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.

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

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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.

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