Acquired Orphan Blood Diseases Therapeutics Market

Acquired Orphan Blood Diseases Therapeutics Market Size:

The global acquired orphan blood diseases therapeutics market size reached USD 9.5 Billion in 2024. Looking forward, IMARC Group expects the market to reach USD 18.1 Billion by 2033, exhibiting a growth rate (CAGR) of 7.13% during 2025-2033. The increasing diagnosis rates of rare blood disorders, advancements in gene therapy and biologics, growing government support through orphan drug designations and regulatory incentives, the rising awareness of these conditions, and improvements in treatment efficacy are some of the major factors propelling the market growth.

Acquired Orphan Blood Diseases Therapeutics Market Analysis:

• Major Market Drivers: The rising prevalence and diagnosis of rare blood disorders, such as acquired hemophilia and paroxysmal nocturnal hemoglobinuria (PNH) represent the major driver of the market. As diagnostic tools improve, more cases are identified, increasing the demand for specialized treatments. Government support through orphan drug designations, financial grants, and extended market exclusivity further incentivizes pharmaceutical companies to invest in developing novel therapies for these rare diseases, making it financially viable for them to address small patient populations. Advancements in gene therapy, biologics, and precision medicine offer promising treatment options, thereby driving market growth.
• Key Market Trends: The growing focus on personalized medicine and targeted therapies represents the key trends in the market. Gene therapies and biologics are gaining traction due to their potential to address the underlying causes of rare blood disorders. Moreover, collaborations between biotech firms and pharmaceutical companies are increasing, accelerating drug development and commercialization. Another significant trend is the emphasis on patient advocacy and awareness, leading to earlier diagnoses and improved access to treatment.
• Geographical Trends: According to the acquired orphan blood diseases therapeutics market overview, North America accounts for the largest region in the market across the globe. Advanced healthcare infrastructure, increasing awareness and diagnosis of rare blood disorders, and significant investment in research and development (R&D) are driving the market growth across the region. Government incentives, such as orphan drug designations and grants, encourage pharmaceutical companies to develop treatments for these rare conditions. Strong advocacy and support networks also play a crucial role in improving patient access to therapies. In line with this, the rise of gene therapies, biologics, and personalized medicine technologies further enhances treatment options, thereby, in turn, enhancing the overall acquired orphan blood diseases therapeutics market value.
• Competitive Landscape: Some of the major market players in the acquired orphan blood diseases therapeutics industry include Alexion Pharmaceuticals Inc. (AstraZeneca plc), Amgen Inc., GSK plc, Novartis AG, Novo Nordisk A/S, Otsuka Pharmaceutical Co. Ltd., Rigel Pharmaceuticals Inc., Roche Holding AG, Sanofi, Takeda Pharmaceutical Company Limited, among many others.
• Challenges and Opportunities: The market faces various challenges including high research and development (R&D) costs, limited patient populations, and complex regulatory pathways. However, the market also faces several opportunities, such as orphan drug status, which offers financial support and extended market exclusivity. Advances in gene therapy, biologics, and precision medicine create new therapeutic possibilities, while growing awareness and improved diagnostics enhance the early detection and treatment, thereby further expanding the market potential.
   

Acquired Orphan Blood Diseases Therapeutics Market Trends:

Rising Prevalence of Rare Blood Disorders

The rise in cases of orphan blood diseases, such as hemophilia, paroxysmal nocturnal hemoglobinuria, and aplastic anemia, has created a growing need for targeted therapies. According to industry reports, India is home to the second-largest population of patients living with Haemophilia, with an estimated 1,36,000 individuals grappling with hemophilia A. However, only around 21,000 are registered at present. Nearly 80 percent of hemophilia cases are undiagnosed in India as several hospitals and medical institutions lack screening capabilities for blood clotting, affecting the diagnosis of new cases. As diagnostic techniques improve, more patients are being diagnosed, thus increasing the demand for specialized treatments, thus creating a positive acquired orphan blood diseases therapeutics market outlook.

Advances in Drug Development

Innovations in gene therapy, biologics, and precision medicine are transforming the treatment landscape for rare blood disorders. For instance, in April 2024, India launched its first home-grown gene therapy for cancer at IIT Bombay. Speaking on the occasion, the President of the country mentioned that the launch of India’s first gene therapy is a breakthrough in the country’s battle against cancer. As this line of treatment, named “CAR-T cell therapy”, is accessible and affordable, it provides new hope for the whole of humankind. The President highlighted that CAR-T cell therapy is one of the most phenomenal advances in medical science. According to the acquired orphan blood diseases therapeutics market revenue, these breakthroughs offer more effective and personalized treatment options, which enhance patient outcomes and drive market growth.

Government Support and Orphan Drug Designations

Regulatory incentives such as orphan drug status, grants, and extended market exclusivity encourage pharmaceutical companies to develop drugs for rare blood diseases. For instance, in August 2024, Opaganib, a proprietary and investigational host-directed drug developed by Tel Aviv-based RedHill Biopharma, was given orphan drug designation by the FDA for the treatment of neuroblastoma, a type of cancer that is the most common malignancy among infants, with a median age of 17 months at the time of diagnosis. RedHill announced the FDA decision in a press release on Aug. 26, 2024. FDA has the authority to grant a drug or biological product “orphan” status if such a product prevents, diagnoses, or treats a rare disease or condition. Neuroblastoma is rare among children overall, accounting for up to 10% of all childhood cancers in the United States, where it affects 11 to 13 children per million under the age of 15—though that number rises to 65 per million under one year old, the equivalent of approximately 650 new cases every year. According to the acquired orphan blood diseases therapeutics market forecast, these incentives make it more financially viable to invest in research and development (R&D), thus contributing to the market's expansion.

Acquired Orphan Blood Diseases Therapeutics Market Segmentation:

IMARC Group provides an analysis of the key trends in each segment of the market, along with forecasts at the global, regional, and country levels for 2025-2033. Our report has categorized the market based on therapy, disease indication, and distribution channel.

Breakup by Therapy:

• Recombinant Factor
• Immunoglobulin Infusion Therapy
• Activated Prothrombin Complex Concentrate
• Thrombopoietin Receptor Agonists
• Others
   

The report has provided a detailed breakup and analysis of the market based on the therapy. This includes recombinant factor, immunoglobulin infusion therapy, activated prothrombin complex concentrate, thrombopoietin receptor agonists, and others.

Recombinant factor therapy is crucial for treating acquired hemophilia, providing a synthetic, non-human-derived clotting factor to control bleeding. Its efficacy in managing bleeding episodes with fewer risks of viral contamination makes it highly valuable in orphan blood disorders, driving its demand in the market, especially as it reduces dependency on plasma-derived products.

Immunoglobulin infusion therapy is widely used to manage autoimmune blood disorders, like acquired hemophilia and immune thrombocytopenia. It helps regulate immune system responses, controlling antibody-mediated destruction of platelets and red blood cells. Its ability to treat both acute and chronic conditions drives demand, making it an essential segment of the acquired orphan blood disease therapeutics market.

Activated prothrombin complex concentrate (aPCC) is vital for patients with hemophilia who develop inhibitors, offering an effective treatment to control bleeding by bypassing the need for the missing clotting factor. Its ability to manage life-threatening bleeds in hemophilia patients with inhibitors makes it a key therapeutic option, driving its demand in the orphan blood diseases market.

Thrombopoietin receptor agonists stimulate platelet production in patients with immune thrombocytopenia (ITP) and aplastic anemia. These drugs are critical for managing chronic low platelet counts, reducing the risk of bleeding complications. Their efficacy in increasing platelet levels in difficult-to-treat cases makes them a driving force in the market for treating rare acquired blood disorders.

Breakup by Disease Indication:

• Acquired Agranulocytosis
• Acquired Hemophilia
• Acquired Von Willebrand Syndrome
• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Myelodysplastic Syndrome
• Others
   

Acquired hemophilia holds the largest share of the industry

A detailed breakup and analysis of the market based on the disease indication has also been provided in the report. This includes acquired agranulocytosis, acquired hemophilia, acquired Von Willebrand syndrome, paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome, and others. According to the report, acquired hemophilia accounted for the largest market share.

Acquired hemophilia accounts for the largest segment in the market due to its severe, life-threatening nature and the need for specialized treatment options. Patients with acquired hemophilia experience spontaneous bleeding episodes, requiring immediate intervention with targeted therapies like recombinant clotting factors, bypassing agents, or immunosuppressive treatments, which is further driving the acquired orphan blood diseases therapeutics demand. The rarity of the disorder, combined with growing awareness and improved diagnostics, drives the demand for effective therapies, making it the largest segment in this niche therapeutic market.

Breakup by Distribution Channel:

• Hospital Pharmacy
• Retail Pharmacy
• Others
   

Hospital pharmacy represents the leading market segment 

A detailed breakup and analysis of the market based on the distribution channel has also been provided in the report. This includes hospital pharmacy, retail pharmacy, and others. According to the acquired orphan blood diseases therapeutics market report, hospital pharmacy accounted for the largest market share.

Hospital pharmacies account for the largest segment in the market due to their critical role in managing complex, severe cases of rare blood disorders. Hospitals provide specialized care and immediate access to advanced treatments, such as recombinant factors and immunoglobulin infusions, which are often required for managing acute and chronic conditions. The high level of patient monitoring and the need for tailored therapy in hospital settings drive the significant demand for these therapeutics, making hospital pharmacies the leading segment.

Breakup by Region:

• North America
  • United States
  • Canada
• Asia Pacific
  • China
  • Japan
  • India
  • South Korea
  • Australia
  • Indonesia
  • Others
• Europe
  • Germany
  • France
  • United Kingdom
  • Italy
  • Spain
  • Russia
  • Others
• Latin America
  • Brazil
  • Mexico
  • Others
• Middle East and Africa
   

North America leads the market, accounting for the largest acquired orphan blood diseases therapeutics market share

The report has also provided a comprehensive analysis of all the major regional markets, which include North America (the United States and Canada); Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others); Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others); Latin America (Brazil, Mexico, and others); and the Middle East and Africa. According to the report, North America represents the largest regional market for acquired orphan blood diseases therapeutics.

In North America, the market is primarily driven by advanced healthcare infrastructure that supports the development and distribution of specialized treatments. Increased awareness and improved diagnostic capabilities lead to higher detection rates of rare disorders, thus driving the demand for targeted therapies. Government incentives, such as orphan drug designations and financial grants, encourage pharmaceutical companies to invest in drug development. Additionally, advancements in gene therapy and biologics offer new, effective treatment options, while strong patient advocacy and support networks enhance access to therapies, further propelling the acquired orphan blood diseases therapeutics market growth. For instance, in August 2023, Regeneron Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has approved Veopoz™ (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE.

Competitive Landscape:

• The market research report has also provided a comprehensive analysis of the competitive landscape in the market. Detailed profiles of all major companies have also been provided. Some of the major market players in the acquired orphan blood diseases therapeutics industry include Alexion Pharmaceuticals Inc. (AstraZeneca plc), Amgen Inc., GSK plc, Novartis AG, Novo Nordisk A/S, Otsuka Pharmaceutical Co. Ltd., Rigel Pharmaceuticals Inc., Roche Holding AG, Sanofi, and Takeda Pharmaceutical Company Limited.
  
  (Please note that this is only a partial list of the key players, and the complete list is provided in the report.)
   
• The market is competitive, with key players such as Alexion Pharmaceuticals, Novartis, and Pfizer leading in drug development for rare blood disorders like paroxysmal nocturnal hemoglobinuria and aplastic anemia. These acquired orphan blood diseases therapeutics companies focus on innovative biologics, gene therapies, and immune-modulating treatments. Smaller biotech firms and startups are also entering the market, leveraging orphan drug designations and regulatory incentives. Competition is driven by advancements in treatment efficacy, pricing strategies, and efforts to secure extended market exclusivity. For instance, in December 2023, Novartis announced that the U.S. Food and Drug Administration (FDA) approved Fabhalta® (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]).

Acquired Orphan Blood Diseases Therapeutics Market News:

• In April 2024, VOYDEYA™ (danicopan) was approved in the US as an add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). VOYDEYA is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant EVH while treated with a C5 inhibitor.
• In February 2023, GSK plc announced that the US Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Benlysta (belimumab), a B-cell inhibiting monoclonal antibody, for the potential treatment of systemic sclerosis. GSK plans to initiate a phase II/III trial of belimumab for systemic sclerosis-associated interstitial lung disease (SSc-ILD) in the first half of 2023.

Acquired Orphan Blood Diseases Therapeutics Market Report Scope:

Key Questions Answered in This Report:

• How has the global acquired orphan blood diseases therapeutics market performed so far, and how will it perform in the coming years?
• What are the drivers, restraints, and opportunities in the global acquired orphan blood diseases therapeutics market?
• What is the impact of each driver, restraint, and opportunity on the global acquired orphan blood diseases therapeutics market?
• What are the key regional markets?
• Which countries represent the most attractive acquired orphan blood diseases therapeutics market?
• What is the breakup of the market based on the therapy?
• Which is the most attractive therapy in the acquired orphan blood diseases therapeutics market?
• What is the breakup of the market based on the disease indication?
• Which is the most attractive disease indication in the acquired orphan blood diseases therapeutics market?
• What is the breakup of the market based on the distribution channel?
• Which is the most attractive distribution channel in the acquired orphan blood diseases therapeutics market?
• What is the competitive structure of the market?
• Who are the key players/companies in the global acquired orphan blood diseases therapeutics market?

Key Benefits for Stakeholders:

• IMARC’s industry report offers a comprehensive quantitative analysis of various market segments, historical and current market trends, market forecasts, and dynamics of the acquired orphan blood diseases therapeutics market from 2019-2033.
• The acquired orphan blood diseases therapeutics market research report provides the latest information on the market drivers, challenges, and opportunities in the global market.
• The study maps the leading, as well as the fastest-growing, regional markets. It further enables stakeholders to identify the key country-level markets within each region.
• Porter's five forces analysis assists stakeholders in assessing the impact of new entrants, competitive rivalry, supplier power, buyer power, and the threat of substitution. It helps stakeholders to analyze the level of competition within the acquired orphan blood diseases therapeutics industry and its attractiveness.
• The competitive landscape allows stakeholders to understand their competitive environment and provides insight into the current positions of key players in the market.