Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) Market Report 2026

The paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market size has grown strongly in recent years. It will grow from $5.97 billion in 2025 to $6.41 billion in 2026 at a compound annual growth rate (CAGR) of 7.3%. The growth in the historic period can be attributed to limited availability of targeted complement therapies, delayed diagnosis due to disease rarity, reliance on plasma exchange and supportive care, high treatment costs restricting access, low awareness of complement-mediated disorders.

The paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market size is expected to see strong growth in the next few years. It will grow to $8.42 billion in 2030 at a compound annual growth rate (CAGR) of 7.1%. The growth in the forecast period can be attributed to development of next-generation complement inhibitors, increasing diagnosis rates through advanced testing, growing focus on orphan disease drug development, expansion of reimbursement coverage for rare diseases, rising establishment of specialty hematology and nephrology centers. Major trends in the forecast period include rising adoption of complement inhibitor therapies, increasing use of genetic and biomarker testing, growth of long-acting complement inhibition drugs, expansion of specialized rare disease treatment centers, improved early diagnosis of complement-mediated disorders.

The rising prevalence of rare diseases is expected to drive the growth of the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market going forward. Rare diseases are conditions that affect a small proportion of the population, generally fewer than 200,000 individuals, and are often marked by clinical complexity, severity, and the need for specialized therapies. The increasing identification of rare diseases is largely attributed to advancements in diagnostic technologies, which allow improved detection and recognition of conditions that were previously underdiagnosed or overlooked. PNH and aHUS contribute to progress in the rare disease landscape by enhancing understanding of disease mechanisms and identifying potential therapeutic targets for disorders with limited treatment options. For example, in November 2024, according to the US Food and Drug Administration, more than 7,000 rare diseases affected over 30 million people in the United States. Therefore, the growing prevalence of rare diseases is driving the expansion of the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market.

Major companies operating in the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market are increasingly focusing on the development of innovative therapies, such as complement inhibitors, to enhance treatment outcomes and improve patient convenience. Complement inhibitors are therapeutic agents that target and block specific components of the complement system, which plays a key role in immune response and inflammation. For instance, in December 2023, Novartis AG, a Switzerland-based pharmaceutical company, received approval from the US Food and Drug Administration for Fabhalta (iptacopan), the first oral monotherapy for treating adults with paroxysmal nocturnal hemoglobinuria. This therapy demonstrated superior improvements in hemoglobin levels without the need for blood transfusions. By inhibiting Factor B in the alternative complement pathway, the drug provides effective control of both intravascular and extravascular hemolysis. Its oral formulation simplifies long-term disease management and enhances patient quality of life by eliminating the need for frequent infusions.

In January 2025, Samsung Bioepis Co. Ltd., a South Korea-based biopharmaceutical company, entered into a partnership with Teva Pharmaceutical Industries Ltd. to develop and commercialize a biosimilar version of Soliris (eculizumab), including EPYSQLI (eculizumab-aagh), for the treatment of rare diseases. This collaboration is intended to expand access to more affordable treatment options for patients in the United States. Teva Pharmaceutical Industries Ltd. is an Israel-based pharmaceutical company engaged in the development, manufacturing, and commercialization of medicines for a range of rare diseases, including paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

Major companies operating in the paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market are Roche Holding AG, Alexion Pharmaceuticals Inc, Genentech Inc, Novartis AG, Apellis Pharmaceuticals Inc, Omeros Corporation, BioCryst Pharmaceuticals Inc, ChemoCentryx Inc, Sanofi SA, CSL Behring, Takeda Pharmaceutical Company, Pfizer Inc, Regeneron Pharmaceuticals Inc, Ionis Pharmaceuticals Inc, Horizon Therapeutics, Achillion Pharmaceuticals Inc, Sobi Swedish Orphan Biovitrum AB, ImmunoForge Co Ltd, Care Pharma, Daiichi Sankyo Co Ltd, Astellas Pharma Inc, Mallinckrodt Pharmaceuticals, CANbridge Pharmaceuticals Inc.

North America was the largest region in the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, 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, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Tariffs have impacted the pnh and atypical hemolytic uremic syndrome market by increasing costs for imported biologic drugs, diagnostic kits, and specialized laboratory equipment used in disease diagnosis and management. Hospital and specialty clinic segments in north america, europe, and asia-pacific are most affected due to dependence on global pharmaceutical supply chains. These cost pressures can delay patient access to advanced therapies. However, tariffs are encouraging domestic biologics manufacturing, regional sourcing of diagnostics, and supply chain diversification, strengthening long-term market resilience.

The paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market research report is one of a series of new reports that provides paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market statistics, including paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) industry global market size, regional shares, competitors with a paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market share, detailed paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market segments, market trends and opportunities, and any further data you may need to thrive in the paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) industry. This paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) 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.

Paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are rare, life-threatening blood disorders caused by uncontrolled activation of the complement system, a component of the immune response. Paroxysmal nocturnal hemoglobinuria (PNH) results in the destruction of red blood cells, whereas atypical hemolytic uremic syndrome (aHUS) leads to the formation of blood clots in small blood vessels, mainly affecting the kidneys and other critical organs.

The main treatment options for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) include Soliris and Ultomiris. Soliris is a monoclonal antibody medication, also known by its generic name eculizumab, used to treat rare and life-threatening blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The disease types addressed include PNH and aHUS, with treatment approaches involving complement inhibitors, plasma exchange or infusion, and other therapies. Diagnosis methods include clinical evaluation, genetic testing, and biomarker testing, and these treatments are used by various end users such as hospitals, specialty clinics, research institutes, and others.

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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