Number of pages: 100 | Report Format: PDF | Published date: March 01, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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.
Paroxysmal Nocturnal Hemoglobinuria Market Fundamentals
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem cell-acquired disease in which red blood cells prematurely break apart. Hematopoietic stem cells are produced in the bone marrow, the soft interior of the body’s long bones. 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 PNH. These mutations cause hematopoietic stem cells to lack glycosyl-phosphatidylinositol anchor protein, resulting in PNH, which can occur at any age. Mostly, the condition affects people of age between 30 and 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.
Paroxysmal Nocturnal Hemoglobinuria Market Dynamics
The increasing prevalence of PNH is the key factor boosting the paroxysmal nocturnal hemoglobinuria market revenue growth. 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 disorder can impact every age group; the thirties represent the median age upon diagnosis.
Increasing research and development for PNH treatment and pathogenesis is a potential factor expected to augment the market revenue growth during the forecast period. For instance, according to 2021 research, the Department of Hematology at Tianjin Medical University suggested new evidence about the pathogenesis of PNH. This evidence provided researchers with a clear picture of the disease, eventually increasing PNH treatment and diagnosis procedures.
The presence of pipeline drugs is also ascribed to an increase in the global paroxysmal nocturnal hemoglobinuria market. For instance, BioCryst Pharmaceuticals is researching BCX9930, a potential drug for PNH. BCX9930 is an oral factor D inhibitor for treating PNH. The global paroxysmal nocturnal hemoglobinuria market revenue growth is also influenced by the presence of several non-profit organizations that help underinsured patients by aiding with their out-of-pocket expenses and fighting for increased access and affordability.
The global paroxysmal nocturnal hemoglobinuria market is likely to 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 healthcare infrastructure and resources and high healthcare costs. This can make it difficult for people to access the healthcare they need.
Paroxysmal Nocturnal Hemoglobinuria Market Ecosystem
The global paroxysmal nocturnal hemoglobinuria market has been analyzed from three perspectives: treatment type, end user, and region.
Paroxysmal Nocturnal Hemoglobinuria Market by Treatment Type
Based on the treatment type, the global paroxysmal nocturnal hemoglobinuria market has been segmented into bone marrow transfusion/stem cell therapy and medications.
The medications segment dominates the global paroxysmal nocturnal hemoglobinuria market. The segment is further sub-segmented into complement inhibitors and monoclonal antibodies. The monoclonal antibodies sub-segment accounts for the majority of the paroxysmal nocturnal hemoglobinuria market revenue share. Monoclonal antibodies optimize the treatment effects of PNH. 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 during the forecast period. The therapy is effective in treating PNH. This treatment has several advantages, including the potential to reduce symptoms, prevent further complications, and improve the 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, the therapy is expected to 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 user, the global paroxysmal nocturnal hemoglobinuria market has been segmented into hospitals and specialty clinics.
The hospitals segment accounts for the majority of the revenue share of the global paroxysmal nocturnal hemoglobinuria market. Hospitals are preferred for treating PNH as they have the necessary equipment and expertise to provide the best possible care. Hospitals have the resources to diagnose PNH accurately and provide the appropriate treatments. Hospitals also enable access to more advanced treatments, such as stem cell transplants, which can treat PNH. Furthermore, hospitals also have the relevant personnel and infrastructure to offer psychological and social support to PNH patients.
Paroxysmal Nocturnal Hemoglobinuria Market by Region
Based on the region, the global paroxysmal nocturnal hemoglobinuria market has been segmented into North America, Europe, Asia Pacific, and the Rest of the World.
North America leads the global market due to the high prevalence of PNH. 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 are anticipated to help in North America paroxysmal nocturnal hemoglobinuria market expansion. 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 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 PNH are driving the revenue growth of the North America paroxysmal nocturnal hemoglobinuria market.
The Europe 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 fueling the Europe paroxysmal nocturnal hemoglobinuria market development. For instance, in June 2020, the European Commission granted orphan drug designation to LNP023 by Novartis Europharm Limited to treat PNH. Furthermore, the number of drug approvals is expected to increase during the forecast period as the prevalence of the disease increases. According to the European Medicines Agency estimates, in 2021, 0.4 out of every 10,000 people had PNH. Awareness related to PNH is also increasing in Europe through webinars and conferences sponsored by market players and support organizations.
The paroxysmal nocturnal hemoglobinuria market in Asia Pacific is expected to expand in terms of revenue share during the forecast period. This can be ascribed to 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 PNH will access new therapies under the Pharmaceutical Benefits Scheme (PBS).
Paroxysmal Nocturnal Hemoglobinuria Market Strategic Developments
Paroxysmal Nocturnal Hemoglobinuria Market Competitive Landscape
The prominent players in the global paroxysmal nocturnal hemoglobinuria market include:
To increase their market revenue share in the paroxysmal nocturnal hemoglobinuria market, leading players are increasingly focusing on implementing strategies, such as adopting recent technologies, product developments, mergers and acquisitions, joint ventures, alliances, and partnerships. They are also expanding their operations in newer regions and improving their presence through acquisitions. Focused research and development efforts enable players to develop innovative products and a broad pipeline of potential new medicines, leading to increase in revenue growth and a strong brand image.
The high prevalence of the disease and advancements in treatment methods are expected to 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.
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