Acquired Orphan Blood Diseases Therapeutics Market – Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Therapy (Recombinant Factor, Immunoglobulin Infusion Therapy, Activated Prothrombin Complex Concentrate, Thrombopoietin Receptor Agonists, Others), By Disease Indication (Acquired Agranulocytosis, Acquired Hemophilia, Acquired Von Willebrand Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplastic Syndrome, Others), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Others), By Region, and Competition 2020-2030F

Market Overview

Global Acquired Orphan Blood Diseases Therapeutics Market has valued at USD 7.95 billion in 2024 and is anticipated to witness an impressive growth in the forecast period with a CAGR of 7.80% through 2030.  Acquired orphan blood diseases is a term that encompasses a group of rare and often life-threatening medical conditions that affect the blood and its components (e.g., red blood cells, white blood cells, platelets) and are not inherited but acquired during a person's lifetime. These blood diseases are not present at birth or inherited through genetics. Instead, they develop or are acquired during a person's life, often due to various triggers or underlying health conditions. Diagnosis, treatment, and management of acquired orphan blood diseases often require specialized care from hematologists or other healthcare providers with expertise in rare blood disorders. Treatment approaches may include blood transfusions, immunosuppressive therapies, targeted therapies, and, in some cases, bone marrow transplantation.

Ongoing advancements in medical research, including genomics, immunology, and molecular biology, have deepened our understanding of the pathogenesis of these diseases. This knowledge supports the development of targeted therapies and treatment innovations. Regulatory incentives provided by orphan drug designations, such as extended market exclusivity, tax credits, and reduced development costs, encourage pharmaceutical companies to invest in research and development for rare diseases. Patient advocacy groups and organizations play a pivotal role in driving research, raising awareness, and advocating for improved access to treatments. Their efforts help accelerate progress in the field of acquired orphan blood diseases. The emergence of novel treatment modalities, including gene therapies, monoclonal antibodies, and immunomodulatory agents, has expanded the range of therapeutic options available for acquired orphan blood diseases. Advances in diagnostic techniques and technologies have led to more accurate and timely diagnoses of acquired orphan blood diseases. This enables early intervention and treatment.

Key Market Drivers

Advancements in Medical Research

Medical research has deepened our understanding of the underlying mechanisms and pathophysiology of acquired orphan blood diseases. This knowledge helps identify novel therapeutic targets and pathways for intervention. For instance, in February 2025, a new 3D lung model will revolutionize respiratory medicine research. This advanced model replicates lung structure and function, enabling more accurate drug testing and disease modeling. It offers a powerful tool for studying conditions like asthma and COPD, accelerating the development of innovative treatments and improving patient outcomes. Research efforts have led to the development of targeted therapies that address the specific molecular and cellular abnormalities associated with these diseases. Targeted treatments often offer better efficacy and safety profiles compared to traditional therapies. Research has contributed to the identification of biomarkers and genetic markers associated with these diseases. Biomarkers enable early diagnosis, disease monitoring, and personalized treatment approaches. Ongoing research supports drug discovery and development programs aimed at creating innovative and more effective therapeutics. This includes the development of monoclonal antibodies, gene therapies, and small molecule drugs. Advances in clinical trial design and methodology, including adaptive trials and innovative endpoints, facilitate the evaluation of potential therapies. Clinical trials are essential for assessing treatment safety and efficacy. Medical research has led to the creation of patient registries and databases for rare diseases. These registries facilitate data collection, epidemiological studies, and clinical trial recruitment.

Collaborative efforts among researchers and healthcare institutions on a global scale promote knowledge sharing and accelerate research progress. These collaborations can lead to the discovery of novel therapies. Research has advanced the concept of precision medicine, allowing for tailored treatment approaches based on individual patient characteristics, including genetics and biomarkers. The development of disease models, such as in vitro cell cultures and animal models, enables researchers to test potential therapies and gain insights into disease mechanisms. Research findings are disseminated through scientific publications and conferences, raising awareness among healthcare providers, patients, and pharmaceutical companies about potential therapeutic options. Medical research actively involves patients in clinical trials and research initiatives. Patient engagement helps ensure that treatments meet the needs and preferences of those affected by these rare diseases. Advances in research often inform regulatory agencies' understanding of rare diseases and their willingness to provide expedited pathways for orphan drug designations and approvals. This factor will help in the development of the Global Acquired Orphan Blood Diseases Therapeutics Market.

Emerging Therapies

Emerging therapies often represent innovative and groundbreaking approaches to treating acquired orphan blood diseases. These therapies may include gene therapies, monoclonal antibodies, cell-based therapies, and small molecule drugs designed to target specific disease mechanisms. For instance, In 2024, Cumberland Pharmaceuticals received FDA Orphan Drug and Rare Pediatric Disease designations for its new Duchenne muscular dystrophy treatment, aiming to address this rare genetic disorder and improve therapeutic options for affected children. Many emerging therapies are designed to be more effective than traditional treatments. They may offer improved disease management, symptom control, and overall outcomes for patients, which makes them highly desirable in the medical community. Some emerging therapies have the potential to modify the course of the disease, halt its progression, or even provide a cure. This transformative impact on patients' lives drives significant demand. Many emerging therapies are designed to be more targeted, which can lead to fewer side effects and a better safety profile compared to older treatments. This can improve patient compliance and overall quality of life. Emerging therapies often take a patient-centric approach, tailoring treatments to individual patients based on their genetic and molecular characteristics. This personalized medicine approach is highly appealing to both patients and healthcare providers. Regulatory agencies may expedite the approval process for emerging therapies, recognizing their potential to address unmet medical needs in rare diseases. This accelerates their availability in the market.

Also, in 2024, high failure rates in sickle cell disease (SCD) treatment development highlight the need for better orphan drug designations and predictive models. Enhanced regulatory support and advanced modeling could improve success rates, accelerating innovative therapies for SCD patients.

The introduction of novel therapies can expand the overall market for acquired orphan blood disease therapeutics as more healthcare providers and patients seek these cutting-edge treatments. The promise of emerging therapies attracts increased investment from pharmaceutical companies and biotechnology firms. This investment drives research and development efforts in the field. Patients and advocacy groups often advocate for access to emerging therapies that can offer better outcomes and quality of life. Their advocacy contributes to the demand for these treatments. As emerging therapies demonstrate their effectiveness, healthcare providers are more likely to adopt them into their treatment protocols, further driving demand. Emerging therapies can lead to better disease management and reduced complications, resulting in cost savings for healthcare systems in the long run. The presence of emerging therapies in the market fosters competition among pharmaceutical companies. This competition can lead to price considerations and potentially more accessible treatment options. This factor will pace up the demand of the Global Acquired Orphan Blood Diseases Therapeutics Market.

Rising Demand of Orphan Drug Designations

Orphan drug designations provide pharmaceutical companies with various incentives, such as extended market exclusivity, tax credits, and research grants. These incentives make it more attractive for companies to invest in the research and development of treatments for rare diseases, including acquired orphan blood diseases. For instance, in 2024, a new CAR T therapy for acute lymphoblastic leukemia (ALL) received FDA Orphan Drug and Rare Pediatric Disease designations. This recognition supports the development of innovative treatments for ALL, a life-threatening cancer, improving options for pediatric and rare disease patients. Regulatory agencies, such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), often offer expedited review and approval processes for orphan drugs. This accelerates the development timeline, allowing promising therapies to reach the market more quickly. Orphan drug designations typically grant a period of market exclusivity during which competing treatments cannot enter the market for the same indication. This exclusivity provides a competitive advantage and encourages pharmaceutical companies to pursue orphan drug development. Tax credits and reduced regulatory fees for orphan drugs can significantly lower the overall cost of drug development. This financial support makes it more financially viable for companies to invest in rare disease therapeutics.

Regulatory incentives often lead to quicker approvals and reduced regulatory burdens, facilitating easier market access for orphan drugs. Healthcare providers and patients can access these therapies sooner, increasing demand. Orphan drug designations attract increased research efforts into acquired orphan blood diseases. This expanded research not only helps identify potential therapies but also raises awareness about these rare conditions. Patient advocacy groups and organizations dedicated to rare diseases actively promote the benefits of orphan drug designations. They advocate for increased funding, research, and access to orphan drugs, contributing to demand. The availability of orphan drugs encourages healthcare providers to improve diagnostic capabilities, leading to earlier and more accurate diagnoses of acquired orphan blood diseases. Timely diagnosis drives the demand for effective therapies. Regulatory agencies worldwide have implemented orphan drug programs and designations, leading to greater international collaboration in rare disease research and development. This harmonization streamlines the path to market for orphan drugs. As more orphan drugs receive designations and approvals, the market for acquired orphan blood disease therapeutics expands. The availability of multiple treatment options contributes to market growth. The prospect of orphan drug designations attracts increased investment from both pharmaceutical companies and venture capitalists. This influx of funding accelerates research and development efforts. Effective orphan drugs can significantly enhance the quality of life for patients with acquired orphan blood diseases. This improvement in patient outcomes drives demand for these treatments. This factor will accelerate the demand of the Global Acquired Orphan Blood Diseases Therapeutics Market.

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Key Market Challenges

Access to Treatment

Rare diseases, including acquired orphan blood diseases, often have a limited number of treatment options available due to their low prevalence. This scarcity of therapies can make it difficult for patients to access appropriate treatments. Many orphan drugs and emerging therapies are associated with high treatment costs. The rarity of these diseases and the costs of research and development often result in expensive therapies, making them financially burdensome for patients and healthcare systems. Acquired orphan blood diseases are not always well-known among healthcare providers and the public. This lack of awareness can lead to delayed diagnoses and difficulty in accessing specialized treatments. Access to advanced medical treatments can vary significantly based on geographic location. Patients in remote or underserved areas may face challenges in accessing specialized healthcare facilities and therapies. In some regions, the healthcare infrastructure may not be adequately equipped to diagnose and manage acquired orphan blood diseases. This can lead to delays in treatment initiation. The availability and extent of insurance coverage for orphan blood disease therapies can vary widely. In some cases, insurance plans may not fully cover the cost of treatment, leaving patients with high out-of-pocket expenses. Pharmaceutical companies may encounter challenges in obtaining reimbursement approvals for orphan drugs, leading to delays in patients' access to treatment. Specialized treatment centers and healthcare providers with expertise in acquired orphan blood diseases may be concentrated in urban areas, making it challenging for patients in rural regions to access care.

Economic and Pricing Pressures

Developing treatments for acquired orphan blood diseases can be exceptionally costly due to the limited patient population, complex research requirements, and the need for specialized therapies. These high development costs can put pressure on pharmaceutical companies to recoup their investments through pricing. The small number of patients with acquired orphan blood diseases means that pharmaceutical companies have a smaller market to target. To make their treatments financially viable, they may need to set higher prices per patient. Pricing for orphan drugs is often scrutinized for transparency, and the public and healthcare providers may question the justification for high drug prices, particularly when the cost-benefit ratio is unclear. Patients and healthcare providers may face challenges in obtaining insurance coverage for expensive orphan drugs. Insurers may hesitate to cover these therapies due to their high cost, leaving patients with significant out-of-pocket expenses. High drug prices can create barriers to access for patients who cannot afford the cost of treatment, potentially limiting their ability to benefit from lifesaving or life-improving therapies. Healthcare systems and payers often have budget constraints, and the introduction of costly orphan drugs can strain healthcare budgets. This can lead to challenges in negotiating pricing agreements with pharmaceutical companies. Economic disparities within and between countries can affect patients' access to orphan drugs. Patients in wealthier regions or countries may have better access, while those in poor areas may struggle to obtain treatment.

Key Market Trends

Expanded Access Programs

(Expanded access programs) EAPs prioritize the needs of patients who have limited or no alternative treatment options. These programs provide access to potentially life-saving therapies, reflecting a patient-centered approach to healthcare. EAPs allow patients to access investigational or emerging therapies that are still in the clinical trial or regulatory approval phases. This can be especially valuable for patients with rare and life-threatening diseases like acquired orphan blood diseases. EAPs are driven by ethical considerations, recognizing the urgency of providing treatments to patients who may not qualify for clinical trials or cannot wait for regulatory approvals. They provide hope and potential benefits to those in need. Patients who do not respond to standard therapies or who have contraindications to traditional treatments may find EAPs as a viable option for accessing novel treatments tailored to their specific condition. EAPs allow for the collection of real-world data and evidence regarding the safety and efficacy of therapies in diverse patient populations. This information can complement clinical trial data and inform treatment decisions. EAPs involve close collaboration between physicians, patients, pharmaceutical companies, and regulatory authorities. This collaboration ensures that patients receive the most appropriate and personalized care.

Segmental Insights

Therapy Insights

In 2024, the Global Acquired Orphan Blood Diseases Therapeutics Market largest share was held by Immunoglobulin Infusion Therapy segment and is predicted to continue expanding over the coming years. Immunoglobulin infusion therapy is a versatile treatment option used for a wide range of acquired orphan blood diseases, including immune thrombocytopenia (ITP), acquired hemophilia, and paroxysmal nocturnal hemoglobinuria (PNH), among others. This broad application makes it a common therapeutic choice for multiple conditions. Immunoglobulin therapy works by modulating the immune system, which is often a key factor in the pathogenesis of acquired orphan blood diseases. It can help regulate autoantibody production and reduce the immune response against self-components, providing a treatment option for autoimmune-based blood disorders. Some acquired orphan blood diseases, such as acquired hemophilia and severe ITP, can lead to life-threatening bleeding episodes. Immunoglobulin infusion therapy can be administered as an emergency treatment to control bleeding, making it a critical option in such situations.

Disease Indication Insights

In 2024, the Global Acquired Orphan Blood Diseases Therapeutics Market largest share was held by Acquired Hemophilia segment and is predicted to continue expanding over the coming years.  Acquired hemophilia is a rare and potentially life-threatening bleeding disorder characterized by the development of autoantibodies against clotting factors, particularly factor VIII. While it is a rare condition, its incidence is relatively higher compared to some other acquired orphan blood diseases, which may contribute to its larger share of the market. Acquired hemophilia can present with severe and spontaneous bleeding episodes, which can be life-threatening. This clinical severity necessitates prompt diagnosis and treatment, leading to a higher demand for therapeutics. Advances in diagnostic methods and increased awareness of acquired hemophilia have led to earlier and more accurate diagnoses. This, in turn, has driven the demand for therapeutic interventions. The treatment of acquired hemophilia can be complex and may require a combination of therapies, including factor replacement, immunosuppressive drugs, and supportive care. These multiple treatment modalities contribute to a larger market share.

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Regional Insights

The North America region dominates the Global Acquired Orphan Blood Diseases Therapeutics Market in 2024. North America, particularly the United States, is a global hub for pharmaceutical and biotechnology research and development. Many leading pharmaceutical companies and research institutions in the region focus on rare diseases, including acquired orphan blood diseases. This concentration of expertise and resources drives innovation and the development of new therapies. The United States has well-established regulatory agencies, such as the U.S. Food and Drug Administration (FDA), that have implemented orphan drug designations and fast-track approval processes for rare disease therapies. These regulatory incentives have encouraged pharmaceutical companies to invest in orphan disease research and development. The North American market, including the United States and Canada, is substantial in terms of both population and healthcare spending. This creates a sizable market for orphan disease therapeutics, attracting investments and driving market growth. North America boasts advanced healthcare infrastructure and facilities, which enable the diagnosis and treatment of rare diseases, including acquired orphan blood diseases. Access to specialized medical centers and expertise is crucial for patients with these conditions.

Recent Developments

• In 2024, AbbVie will acquire Aliada Therapeutics for $1.4bn to enhance its neuroscience pipeline. The deal grants access to Aliada’s blood-brain barrier-crossing technology, including ALIA-1758, a phase 1 anti-pyroglutamate amyloid beta antibody for Alzheimer’s. With 982,000 dementia cases in the UK, Alzheimer’s remains the leading cause, impairing memory, cognition, and daily functions.
• In January 2025, Vertex will pay Orna $65M to develop next-generation gene therapies for blood disorders. This collaboration aims to leverage Orna’s innovative RNA-based technology to advance treatments, potentially improving outcomes for patients with genetic blood conditions through cutting-edge therapeutic approaches.
• In October 2022, Pfizer Inc. has successfully finalized its acquisition of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company with a strong focus on discovering, developing, and delivering transformative treatments, particularly for underserved patient populations, beginning with sickle cell disease (SCD). This acquisition underscores Pfizer's dedication to addressing SCD and further strengthens its position in the rare hematology sector, building upon a 30-year history in this field. GBT comes with a portfolio and pipeline that holds the potential to address the comprehensive requirements of this marginalized community. Among GBT's achievements is the discovery and development of Oxbryta (voxelotor), a pioneering medication that directly targets the root cause of SCD. Furthermore, GBT boasts a promising lineup of preclinical and clinical investigational assets focused on SCD, including GBT021601 (GBT601) and inclaclumab, both of which have earned Orphan Drug and Rare Paediatric Disease designations from the U.S. Food and Drug Administration (FDA).
• In February 2019, he U.S. Food and Drug Administration granted approval to Cablivi (caplacizumab-yhdp) injection. It is the first therapy indicated for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults. Cablivi is approved for use in combination with plasma exchange and immunosuppressive therapy for this rare and life-threatening disorder characterized by abnormal blood clotting. Individuals diagnosed with a TTP experience the formation of widespread blood clots within the body's small blood vessels. These clots have the potential to obstruct oxygen and blood delivery to vital organs, resulting in strokes and heart attacks that can result in brain damage or even fatality. The development of a TTP can be triggered by factors such as cancer, HIV, pregnancy, lupus, infections, or following medical procedures like surgery, bone marrow transplantation, or chemotherapy.

Key Market Players

• Alexion Pharmaceuticals, Inc.
• Amgen, Inc.
• Celgene Corporation
• Eli Lilly and Company
• Sanofi S.A.
• GlaxoSmithKline plc,
• Cyclacel Pharmaceuticals, Inc.
• Onconova Therapeutics, Inc.
• Incyte Corporation,
• CTI BioPharma Corp

Report Scope:

In this report, the Global Acquired Orphan Blood Diseases Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

•     Acquired Orphan Blood Diseases Therapeutics Market, By Therapy:

o   Recombinant Factor

o   Immunoglobulin Infusion Therapy

o   Activated Prothrombin Complex Concentrate

o   Thrombopoietin Receptor Agonists

o   Others

•   Acquired Orphan Blood Diseases Therapeutics Market, By Disease Indication:

o   Acquired Agranulocytosis

o   Acquired Hemophilia

o   Acquired Von Willebrand Syndrome

o   Paroxysmal Nocturnal Hemoglobinuria (PNH)

o   Myelodysplastic Syndrome

o   Others

•     Acquired Orphan Blood Diseases Therapeutics Market, By Distribution Channel:

o   Hospital Pharmacy

o   Retail Pharmacy

o   Others

•    Acquired Orphan Blood Diseases Therapeutics Market, By region:

o   North America

§  United States

§  Canada

§  Mexico

o   Asia-Pacific

§  China

§  India

§  South Korea

§  Australia

§  Japan

o   Europe

§  Germany

§  France

§  United Kingdom

§  Spain

§  Italy

o   South America

§  Brazil

§  Argentina

§  Colombia

o   Middle East & Africa

§  South Africa

§  Saudi Arabia

§  UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Acquired Orphan Blood Diseases Therapeutics Market.

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Company Information

• Detailed analysis and profiling of additional market players (up to five).

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