DelveInsight's "Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Paroxysmal Nocturnal Hemoglobinuria, historical and forecasted epidemiology as well as the Paroxysmal Nocturnal Hemoglobinuria market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The Paroxysmal Nocturnal Hemoglobinuria market report provides current treatment practices, emerging drugs, Paroxysmal Nocturnal Hemoglobinuria market share of the individual therapies, and current and forecasted Paroxysmal Nocturnal Hemoglobinuria market Size from 2019 to 2032 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 of the opportunities and assesses the underlying potential of the Paroxysmal Nocturnal Hemoglobinuria market.
Some of the key facts of the Paroxysmal Nocturnal Hemoglobinuria Market Report:
- According to George et al., The median age of diagnosis is 35-40 years of age, with occasional cases diagnosed in childhood or adolescence
- As per George et al., the risk of developing PNH after treatment for aplastic anemia with immunosuppressive therapy (anti-thymocyte globulin and cyclosporine) is approximately 20-30%.
Key Benefits of the Report:
- The report covers the descriptive overview of Paroxysmal Nocturnal Hemoglobinuria, explaining its causes, signs and symptoms, pathophysiology, diagnosis, and currently available therapies
- Comprehensive insight has been provided into the Paroxysmal Nocturnal Hemoglobinuria epidemiology and treatment in the 7MM
- Additionally, an all-inclusive account of both the current and emerging therapies for Paroxysmal Nocturnal Hemoglobinuria is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
- A detailed review of the Paroxysmal Nocturnal Hemoglobinuria market; historical and forecasted is included in the report, covering drug outreach in the 7MM
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Paroxysmal Nocturnal Hemoglobinuria market
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Paroxysmal Nocturnal Hemoglobinuria Overview
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood. This disease is due to mutation of the X-linked gene phosphatidylinositol glycan class A (PIGA), leading to deficiency in the glycosylphosphatidylinositol (GPI) protein, which is responsible for anchoring other protein moieties to the surface of erythrocytes.
The appropriate treatment for PNH depends on the severity of the symptoms. Some patients will experience few or no symptoms from PNH and do not require treatment other than folic acid and sometimes iron supplementation to increase red blood cell production. For symptomatic PNH patients, the mainstay treatment is the drug eculizumab (Soliris). It is a monoclonal antibody that inhibits C5 in the terminal complement cascade, interrupting RBC destruction. It reduces the risk of thrombosis and improves the quality of life in PNH patients. The treatment landscape of PNH is highly active with recent launches of approved drugs. In May 2021, US FDA approved Empaveli (pegcetacoplan) injection to treat adults with PNH. Empoli is the first PNH treatment that binds to complement protein C3. Ultomiris, a drug approved in 2018 for the treatment of PNH in adults, gained expanded access to include children (one month of age and older) and adolescents with PNH, in June 2021. Ultomiris is the first and only medicine approved in US to treat children and adolescents with PNH. Both of these drugs were given priority review by US FDA.
Paroxysmal Nocturnal Hemoglobinuria Epidemiological Insights
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease, with a prevalence estimated between 0.5-1.5 per million people in the general population. PNH is believed to affect males and females in equal numbers, although some studies show a slight female preponderance. The condition is genetic, with the mutations occurring on the X-linked gene. As PNH presents with a variety of symptoms, few patients don’t get diagnosed until later stages.
- According to Nishimura et al., the occurrence of PNH has no apparent ethnic or geographic distribution, however, about 30-40% of PNH cases are reported in the United States and Europe, whereas <10% of PNH cases are reported from Asia.
- According to Kanakura et al., thrombosis is the main reason for severe complications and mortality in PNH and it is found that around 40% percent of PNH patients develop thrombosis at some point in their disease course. The incidence of thromboembolism due to PNH is higher in the US and Europe as compared to Japan.
- According to NORD, the disorder can affect any age group. The median age of diagnosis is during the 30s. Most populations are in between 30-40 years of age.
Paroxysmal Nocturnal Hemoglobinuria Epidemiological Segmentation
- Paroxysmal Nocturnal Hemoglobinuria total prelevant cases
- Paroxysmal Nocturnal Hemoglobinuria total diagnosed cases
- Paroxysmal Nocturnal Hemoglobinuria age specific cases
Paroxysmal Nocturnal Hemoglobinuria Market Outlook
The Paroxysmal Nocturnal Hemoglobinuria market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Paroxysmal Nocturnal Hemoglobinuria market trends by analyzing the impact of current Paroxysmal Nocturnal Hemoglobinuria therapies on the market, unmet needs, drivers and 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 2019-2032.
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Paroxysmal Nocturnal Hemoglobinuria Key Companies
- Novartis
- Apellis Pharma
- Novel Med
- and many more
Paroxysmal Nocturnal Hemoglobinuria Therapies
- LNP023
- NM8074
- APL-2
- and many more
Table of Contents
- Key Insights
- Report Introduction
- Executive Summary of Paroxysmal Nocturnal Hemoglobinuria
- Disease Background and Overview
- Epidemiology and patient population
- The United States
- EU 5
- Paroxysmal Nocturnal Hemoglobinuria Emerging Therapies
- Paroxysmal Nocturnal Hemoglobinuria Market Outlook
- Market Access and Reimbursement of Therapies
- Appendix
- Paroxysmal Nocturnal Hemoglobinuria Report Methodoloy
- DelveInsight Capabilities
- Disclaimer
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