Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Global Market Report 2025

The growing prevalence of rare diseases is expected to drive the growth of the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market. Rare diseases are medical conditions that affect a small percentage of the population, typically fewer than 200,000 individuals, and are often characterized by their complexity, severity, and the need for specialized treatment. The rise in rare diseases can be attributed to advances in diagnostic technologies, which have improved the detection and identification of previously underreported or undiagnosed conditions. PNH and aHUS contribute to a deeper understanding and treatment of rare diseases by providing insights into their pathophysiology and potential therapeutic targets for conditions with limited treatment options. For example, in November 2024, the Food and Drug Administration (FDA) reported that over 7,000 rare diseases affected more than 30 million individuals in the United States. As a result, the increasing prevalence of rare diseases is driving market growth in the PNH and aHUS sectors.

Leading companies in the PNH and aHUS market are focusing on the development of innovative therapies, such as complement inhibitors, to enhance treatment outcomes and patient convenience. Complement inhibitors are therapeutic agents that block specific components of the complement system, which plays a role in clearing pathogens and damaged cells. For example, in December 2023, Novartis AG, a pharmaceutical company based in Switzerland, received FDA approval for Fabhalta (iptacopan), the first oral monotherapy for treating adults with paroxysmal nocturnal hemoglobinuria. Fabhalta offers superior hemoglobin improvement without the need for transfusions. This innovative complement inhibitor works by inhibiting Factor B in the alternative complement pathway, effectively controlling both intravascular and extravascular hemolysis. Its oral administration simplifies disease management and improves the quality of life for patients, eliminating the burden of frequent infusions.

In January 2025, Samsung Bioepis Co. Ltd., a biopharmaceutical company based in South Korea, partnered with Teva Pharmaceutical Industries Ltd. to develop and commercialize Soliris (eculizumab) biosimilars, including EPYSQLI (eculizumab-aagh), for rare diseases. This collaboration aims to improve access to affordable treatment options for U.S. patients. Teva, an Israeli pharmaceutical company, specializes in the development, manufacturing, and commercialization of medicines for various rare diseases, including PNH and aHUS.

Major players in the paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market are Roche Holding AG, AstraZeneca plc, Novartis AG, Amgen Inc., Teva Pharmaceuticals Ltd., Regeneron Pharmaceuticals Inc., Chugai Pharmaceutical Co. Ltd., Alexion Pharmaceuticals Inc., UCB Pharma Limited, Alnylam Pharmaceuticals Inc., BioCryst Pharmaceuticals Inc., Genentech Inc., Omeros Corporation, CinnaGen Co., Apellis Pharmaceuticals Inc., CANbridge Pharmaceuticals Inc., Kira Pharmaceuticals, NovelMed Therapeutics Inc., Arrowhead Pharmaceuticals Inc., Akari Therapeutics plc.

North America was the largest region in the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market in 2024. Asia Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) 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) and atypical hemolytic uremic syndrome (aHUS) market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market consists of revenues earned by entities by providing services such as bone marrow biopsy, flow cytometry testing, and multidisciplinary care coordination. 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) and atypical hemolytic uremic syndrome (aHUS) market also includes sales of flow cytometry reagents, anticoagulants, and pegcetacoplan. 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.

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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