Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Report 2025

This report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.

The paroxysmal nocturnal hemoglobinuria (PNH) market size has grown rapidly in recent years. It will grow from $4.24 billion in 2024 to $4.68 billion in 2025 at a compound annual growth rate (CAGR) of 10.4%. The growth in the historic period can be attributed to growing demand for paroxysmal nocturnal hemoglobinuria treatment, rising disposable incomes, government initiatives, rising healthcare expenditure.

The paroxysmal nocturnal hemoglobinuria (PNH) market size is expected to see strong growth in the next few years. It will grow to $6.69 billion in 2029 at a compound annual growth rate (CAGR) of 9.3%. The growth in the forecast period can be attributed to aging population, growing demand for self-administered treatments, increasing access to healthcare, increasing prevalence of comorbidities, growing demand for personalized medicine. Major trends in the forecast period include digital health technologies, development of new and innovative treatments,adoption of gene therapy, early intervention.

The growing prevalence of blood disorders and bone marrow-related conditions is expected to drive the growth of the paroxysmal nocturnal hemoglobinuria (PNH) market. Blood disorders are conditions that affect the production, function, and composition of blood and its components, while bone marrow-related disorders involve insufficient production of red, white blood cells, or platelets, or the production of damaged or defective blood cells. PNH, a rare blood disorder linked to bone marrow failure, occurs when a genetic mutation disrupts the function of red blood cells and platelets. For example, in August 2022, Cancer Australia reported that around 5,202 new cases of leukemia were expected in 2022, with 3,198 cases in males and 2,004 cases in females. The likelihood of being diagnosed with leukemia by age 85 is 1 in 58 (1.7%) overall, with males at a higher risk (1 in 47 or 2.1%) compared to females (1 in 77 or 1.3%). This increasing incidence of blood and bone marrow disorders is driving the growth of the PNH market.

The growing requirement for blood transfusions is poised to be a driving force behind the expansion of the paroxysmal nocturnal hemoglobinuria (PNH) market. Blood transfusion is a medical procedure that involves the transfer of blood or blood products from a donor to a recipient. For individuals with paroxysmal nocturnal hemoglobinuria (PNH), blood transfusions can offer temporary relief from the symptoms and complications associated with this condition. For instance, in June 2023, the World Health Organization, an intergovernmental organization headquartered in Switzerland, reported that high-income nations, which account for only 16% of the global population, contributed significantly to blood donations, comprising 40% of the total 118.5 million blood donations made worldwide. Additionally, in December 2022, the Government of Canada reported that the United Kingdom experienced an upsurge in blood donations, with 1.7 million donations made across the country in 2021. Consequently, the escalating demand for blood transfusions is a key factor driving the growth of the paroxysmal nocturnal hemoglobinuria (PNH) market.

Leading companies in the paroxysmal nocturnal hemoglobinuria (PNH) market are focusing on the development of innovative therapies, such as injectable C5 inhibitors, to maintain their competitive edge. Injectable C5 inhibitors are monoclonal antibodies that block complement component C5, helping to reduce inflammation and tissue damage associated with autoimmune diseases, including PNH. For example, in February 2024, Chugai Pharmaceutical Co., a Japan-based pharmaceutical company, received priority review approval from China's National Medical Products Administration (NMPA) for its PNH treatment. This makes China the first country to approve PIASKY, allowing it to enter negotiations for the National Reimbursement Drug List (NRDL) within the same year. PNH, a rare disease, affects an estimated 12,740 individuals in China, presenting substantial market opportunities.

In March 2024, Gilead Sciences, Inc., a US-based biopharmaceutical company specializing in developing medicines for life-threatening diseases, acquired Cymabay Therapeutics, Inc. for $4.3 billion. This acquisition strengthens Gilead's liver disease portfolio, particularly with the addition of seladelpar, a promising treatment for primary biliary cholangitis that addresses significant unmet medical needs. Cymabay Therapeutics, Inc., also based in the US, is focused on developing seladelpar as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH).

Major companies operating in the paroxysmal nocturnal hemoglobinuria (pnh) market are Pfizer Inc., Johnson & Johnson Pvt Ltd., F. Hoffmann-La Roche AG., Novartis AG, Sanofi S.A., Thermo Fisher Scientific Inc., Takeda Pharmaceutical Company Limited, Amgen Inc., GE Healthcare, Regeneron Pharmaceuticals Inc., CSL Behring LLC, Biogen Inc., Vertex Pharmaceuticals Incorporated, Grifols S.A., Genentech Inc., Shire PLC, Alnylam Pharmaceuticals Inc., Innovent Biologics Inc., Bioverativ Inc., CinnaGen Co, BioCryst Pharmaceuticals, Omeros Corporation, Hansa Biopharma AB, Apellis Pharmaceuticals Inc., Kira Pharmaceuticals Ltd., Akari Therapeutics PLC, RA Pharmaceuticals Inc., C5 Therapeutics Inc.

North America was the largest region in the paroxysmal nocturnal hemoglobinuria (PNH) market in 2024. The regions covered in paroxysmal nocturnal hemoglobinuria (PNH) report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the paroxysmal nocturnal hemoglobinuria (PNH) market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The paroxysmal nocturnal hemoglobinuria (PNH) market consists of revenues earned by entities by providing services such as gene therapy, stem cell transplant and complete blood count test (CBC). The market value includes the value of related goods sold by the service provider or included within the service offering. The paroxysmal nocturnal hemoglobinuria (PNH) market also includes sales of Eculizumab and Ravulizumab. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

Paroxysmal nocturnal hemoglobinuria (PNH) is an uncommon hematological disorder that is acquired, and it is characterized by the abnormal breakdown of red blood cells (hemolysis) due to a deficiency of certain proteins found on the surface of blood cells. This condition is debilitating and can lead to premature death, as well as impaired blood cell production. PNH manifests through a variety of symptoms, including fatigue, shortness of breath, abdominal pain, and the formation of blood clots known as thrombosis.

The primary approaches to treating paroxysmal nocturnal hemoglobinuria (PNH) involve the use of medications, bone marrow transplantation, blood transfusion, immunosuppressive therapy, and other methods. Medications are substances specially designed and formulated for the diagnosis, treatment, prevention, or alleviation of medical conditions, diseases, or symptoms in both humans and animals. Various diagnostic techniques, such as flow cytometry and high-resolution mass spectrometry, are utilized by a range of end users, including hospitals, clinics, and others.

The paroxysmal nocturnal hemoglobinuria market research report is one of a series of new reports that provides paroxysmal nocturnal hemoglobinuria market statistics, including paroxysmal nocturnal hemoglobinuria industry global market size, regional shares, competitors with a paroxysmal nocturnal hemoglobinuria market share, detailed paroxysmal nocturnal hemoglobinuria market segments, market trends and opportunities and any further data you may need to thrive in the paroxysmal nocturnal hemoglobinuria industry. This paroxysmal nocturnal hemoglobinuria market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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