Paroxysmal Nocturnal Hemoglobinuria Market
Number of pages: 100 | Report Format: PDF | Published date: March 01, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2022-2030
|
Report Attribute |
Details |
|
Market size value in 2023 |
US$ 3.32 billion |
|
Revenue forecast in 2031 |
US$ 7.54 billion |
|
Growth Rate |
CAGR of 10.76% from 2023 to 2031 |
|
Base year for estimation |
2022 |
|
Forecast Period |
2023-2031 |
|
Historical Year |
2021 |
|
Segments Covered |
Treatment Type, and End User. |
|
Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global paroxysmal nocturnal hemoglobinuria market was valued at US$ 3.32 billion in 2023 and is expected to register a revenue CAGR of 10.76% to reach US$ 7.54 billion by 2031.
Market Fundamentals
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem cell-acquired disease in which red blood cells prematurely break apart. In the bone marrow, the soft interior of the body's long bones, hematopoietic stem cells are produced. As they expand, these cells turn into red blood cells, white blood cells, and platelets. People with PNH have certain hematopoietic stem cell defects, which result in defective blood cell production. These PNH red blood cells are especially vulnerable to premature destruction by the complement system of the body's immune system. Somatic mutations in the PIGA gene, which encodes phosphatidylinositol glycan class A, have been linked to the pathogenesis of paroxysmal nocturnal hemoglobinuria (PNH). These mutations cause hematopoietic stem cells to lack glycosyl-phosphatidylinositol anchor protein (GPI-AP), resulting in paroxysmal nocturnal hemoglobinuria. Paroxysmal nocturnal hemoglobinuria (PNH) can occur at any age. Mostly, the condition affects people between 30 to 40. Women are slightly more prone to acquire the disease than men.
PNH symptoms arise from both insufficient blood cell synthesis by the bone marrow and the generation of defective blood cells. Each experiences the condition differently, including its unique symptoms and rate of advancement. Some people may experience minor symptoms that are stable for years, such as headaches, short breath, and bruising easily, while others may experience more severe symptoms that can lead to life-threatening consequences such as blood clots in the veins.
[575676]
Market Dynamics
The increasing prevalence of paroxysmal nocturnal hemoglobinuria is the key factor boosting the paroxysmal nocturnal hemoglobinuria market revenue. According to the National Organization for Rare Diseases, in the general population, the prevalence is predicted to range from 0.5 to 1.5 per million people. The condition has been described in people from a wide range of ethnic origins and has been found everywhere in the world. Those from Southeast Asia or the Far East with higher incidences of aplastic anemia may develop the illness more frequently. The illness can impact every age group; the thirties represent the median age upon diagnosis.
Increasing research and development for paroxysmal nocturnal hemoglobinuria treatment and pathogenesis is a potential factor augmenting the market revenue growth in the forecast period. For instance, according to research in 2021, the Department of Hematology, Tianjin Medical University suggested new evidence about the pathogenesis of paroxysmal nocturnal hemoglobinuria. This evidence gave researchers a clear picture of the disease, eventually increasing paroxysmal nocturnal hemoglobinuria treatment and diagnosis procedures.
The presence of pipeline drugs in the market is also ascribed to an increase in the global paroxysmal nocturnal hemoglobinuria market. For instance, BioCryst Pharmaceuticals is researching BCX9930, a potential drug for paroxysmal nocturnal hemoglobinuria. BCX9930 is an oral factor D inhibitor for treating paroxysmal nocturnal hemoglobinuria. The global paroxysmal nocturnal hemoglobinuria market revenue growth is also influenced by the presence of several non-profit organizations that help underinsured patients by helping with their out-of-pocket expenses and fighting for increased access and affordability.
The global paroxysmal nocturnal hemoglobinuria market will experience limited revenue growth in the middle- and low-income countries such as the Middle East and Africa, where healthcare standards are not advanced. This is mainly due to a lack of infrastructure and resources and high healthcare costs. This can make it difficult for people to access the healthcare they need.
Market Ecosystem
The global paroxysmal nocturnal hemoglobinuria market has been analyzed from four perspectives: treatment type, and end user.
Paroxysmal Nocturnal Hemoglobinuria Market by Treatment Type
[785464]
Based on treatment type, the global paroxysmal nocturnal hemoglobinuria market has been segmented into bone marrow transfusion/stem cell therapy, and medications.
The medications segment has dominated the global paroxysmal nocturnal hemoglobinuria market. The segment is further categorized into complement inhibitors and monoclonal antibodies. The monoclonal antibodies segment has the majority of paroxysmal nocturnal hemoglobinuria market revenue share. Monoclonal antibodies optimize the treatment effects of paroxysmal nocturnal hemoglobinuria. Treatment with monoclonal antibodies is also preferred due to their low toxicity and high specificity effects. Eculizumab, the first anti-C5 monoclonal antibody authorized for PNH, changes the course of the disease by preventing intravascular hemolysis and lowering the risk of thromboembolic events, leading to a considerable improvement in survival and quality of life.
The bone marrow transfusion/ stem cell therapy segment is anticipated to show high revenue growth in the forecast period. The therapy is effective in treating paroxysmal nocturnal hemoglobinuria (PNH). This treatment has several advantages, including the potential to reduce symptoms, prevent further complications, and improve quality of life with PNH. The stem cells used in this therapy are collected from the patient’s bone marrow, so the risk of rejection is low. Furthermore, the cells are treated with antibiotics before infusion, decreasing the risk of rejection. Furthermore, it is expected that the therapy will advance in the coming years as more research and development reveals evidence of its efficacy in treating the disease.
Paroxysmal Nocturnal Hemoglobinuria Market by End User
Based on end users, the global paroxysmal nocturnal hemoglobinuria market has been segmented into hospitals and specialty clinics.
The hospitals segment has the majority of the global paroxysmal nocturnal hemoglobinuria market revenue share. Hospitals are preferred for treating paroxysmal nocturnal hemoglobinuria (PNH) because they have the necessary equipment and expertise to provide the best possible care. Hospitals have the resources to diagnose paroxysmal nocturnal hemoglobinuria accurately and provide the appropriate treatments. Hospitals also enable access to more advanced treatments, such as stem cell transplants, which can treat paroxysmal nocturnal hemoglobinuria. Furthermore, hospitals also have the relevant personnel and infrastructure to provide psychological and social support to PNH patients.
Paroxysmal Nocturnal Hemoglobinuria Market by Region
Based on region, the global paroxysmal nocturnal hemoglobinuria market has been segmented into North America, Europe, Asia Pacific, and the Rest of the World.
North America currently leads the global market because of the high prevalence of paroxysmal nocturnal hemoglobinuria. According to the National Institute of Health, in 2020, the prevalence was estimated at 8 per million and the incidence at about 1 per million. Additionally, a large concentration of market players and increased drug approvals is anticipated to increase North America's global paroxysmal nocturnal hemoglobinuria market. For instance, in December 2022, Swedish Orphan Biovitrum (Sobi) affiliate Sobi North America's Empaveli (pegcetacoplan) was approved by Health Canada to treat people with paroxysmal nocturnal hemoglobinuria (PNH). Patients with inadequate response to intolerance to a C5 inhibitor are recommended with this medication. Moreover, increasing healthcare standards and rising awareness related to paroxysmal nocturnal hemoglobinuria are factors driving the revenue growth of North America paroxysmal nocturnal hemoglobinuria market.
European region for paroxysmal nocturnal hemoglobinuria market is characterized by increasing healthcare standards offering better diagnosis and treatment. Moreover, the rising approval of orphan drugs in the market is also increasing Europe's global paroxysmal nocturnal hemoglobinuria market. For instance, in June 2020, the European Commission granted orphan drug designation to LNP023 by Novartis Europharm Limited to treat paroxysmal nocturnal hemoglobinuria. Furthermore, it is anticipated that drug approvals will increase in the forecast period as the prevalence of the disease will also increase. According to the European Medicines Agency estimates, in 2021, 0.4 out of every 10,000 people had paroxysmal nocturnal hemoglobinuria. Awareness related to paroxysmal nocturnal hemoglobinuria is also increasing in Europe through webinars and conferences sponsored by market players and support organizations.
The global paroxysmal nocturnal hemoglobinuria market in Asia Pacific will increase in the forecast period. This is due to the increased clinical trials, increasing research and development on the disease, and rising healthcare standards. The rising prevalence rate in Asia pacific is increasing the diagnostic and treatment in this region. Furthermore, the global paroxysmal nocturnal hemoglobinuria market will experience revenue growth in the forecast period as the region’s healthcare is gaining advancements in the treatment of PNH. For instance, according to the Department of Health and Aged Care, Australians with paroxysmal nocturnal hemoglobinuria will access new therapies under the Pharmaceutical Benefits Scheme (PBS).
Strategic development
Competitive Landscape
Some prominent market players in the global paroxysmal nocturnal hemoglobinuria market include:
To increase their market position in the paroxysmal nocturnal hemoglobinuria market, the leading players are increasingly focusing on implementing tactics such as adopting recent technologies, product developments, mergers and acquisitions, joint ventures, alliances, and partnerships. The companies are growing their operations and improving their presence through acquisitions. Focused research and development efforts (R&D) enable the players to develop innovative products and a broad pipeline of potential new medicines, leading to revenue growth and helping secure brand image.
The high prevalence of the disease and advancements in treatment methods will drive the global paroxysmal nocturnal hemoglobinuria market.
Some prominent market players in the global paroxysmal nocturnal hemoglobinuria market include Novartis AG, AbbVie Inc., Takeda Pharmaceuticals, ad Mylan N.V. and others.
The medications (complement inhibitors and monoclonal antibodies) segment is anticipated to grow rapidly during the forecast period.
Asia Pacific is expected to mark the highest revenue growth during the forecast period.
The high cost of products and stringent regulations are the most prominent restraints in the global paroxysmal nocturnal hemoglobinuria market.
*Insights on financial performance are subject to the availability of information in the public domain