Global Acquired Orphan Blood Diseases Therapeutics Market Report and Forecast 2025-2034

Global Acquired Orphan Blood Diseases Therapeutics Market Outlook

The global acquired orphan blood diseases therapeutics market size is expected to grow at a CAGR of 7.5% during the forecast period of 2025-2034, driven by the emergence of novel therapies and rising healthcare expenditure across the globe.

Global Acquired Orphan Blood Diseases Therapeutics Market Overview

Acquired orphan blood diseases therapeutics comprise medical treatments specifically designed to treat rare blood conditions. Several diseases such as acquired agranulocytosis, acquired hemophilia and paroxysmal nocturnal hemoglobinuria (PNH) come under the umbrella term of acquired orphan blood diseases. The rising demand for effective treatment solutions for these diseases has resulted in growing research activities, aimed at understanding the prognosis of rare bleeding disorders.

With rare diseases affecting over 30 million Americans , the acquired orphan blood diseases therapeutics demand is expected to witness a notable rise. To address the unmet medical needs of patients with orphan diseases including blood disorders, various government and non-government organizations have been proactive in launching initiatives and programs to accelerate the drug development process. In addition, the acquired orphan blood diseases therapeutics sector is experiencing major funding to support the advancement of innovative therapies. Moreover, key biopharmaceutical companies are entering into strategic partnerships to develop and commercialize clinically effective therapeutics in the international markets.

The market is also driven by the surge in drug approvals by health regulatory bodies related to orphan disease therapeutics, increased healthcare expenditure, and rising disposable incomes. Furthermore, the heightened awareness among patients along with rapid advancements in medical research is also anticipated to fuel the acquired orphan blood diseases therapeutics share in the coming years.

Rise in Drug Approvals

One of the significant market trends observed in the acquired orphan blood diseases therapeutics segment is the surge in drug approvals from health regulatory agencies, such as the United States Food and Drug Administration (FDA). In December 2023 , the FDA approved Novartis AG’s (Swiss multinational pharmaceutical corporation) iptacopan, the first oral monotherapy treatment for adults suffering from paroxysmal nocturnal hemoglobinuria (PNH). This oral medication, sold under the brand name Fabhalta, is expected to address the medical needs of patients dependent on blood transfusions and dealing with anemia despite anti-C5 treatments. The FDA approved Fabhalta based on the positive results of the phase III APPLY-PNH trial, wherein the patients exhibited higher hemoglobin levels compared to anti-C5 treatment. The FDA and European Medicines Agency have also granted orphan drug designations to Fabhalta for PNH.

Intensive Research Activities Boosting Market Growth

Robust research efforts have resulted in the emergence of innovative therapies in the rare disease space, which has fuelled the acquired orphan blood diseases therapeutics growth. In October 2023 , a study published in the Lancet revealed the effectiveness of emicizumab prophylaxis (a humanized, bispecific, monoclonal antibody administered subcutaneously) in preventing bleeding in patients suffering from acquired hemophilia A. The standard treatment for people with this acquired orphan blood disease is immunosuppressive therapy which has exhibited adverse effects and even mortality in certain cases, which necessitated the demand for alternative therapeutics. This research study has suggested that immunosuppressive therapy can be deferred with the help of emicizumab prophylaxis treatment. It was reported that the mean bleeding rate with emicizumab was 0.04 bleeds per patient per week, as compared to 0.15 bleeds observed during immunosuppression therapy, suggesting promising results for the therapeutic antibody.

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Purchase the Regional Overview for USD 1099

If you choose to purchase the full report at a later stage, the amount already paid will be adjusted, and you will only need to pay the remaining balance.

Purchase the Regional Overview for USD 1099

If you choose to purchase the full report at a later stage, the amount already paid will be adjusted, and you will only need to pay the remaining balance.

Purchase the Regional Overview for USD 1099

If you choose to purchase the full report at a later stage, the amount already paid will be adjusted, and you will only need to pay the remaining balance.

Global Acquired Orphan Blood Diseases Therapeutics Market Segmentation

Market Breakup by Therapy

• Recombinant Factor

• Immunoglobulin Infusion Therapy

• Thrombopoietin Receptor Agonists

• Activated Prothrombin Complex Concentrate

• Others

Market Breakup by Indication

• Acquired Agranulocytosis

• Acquired Hemophilia

• Acquired Von Willebrand Syndrome

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Myelodysplastic Syndrome

• Others

Market Breakup by Distribution Channel

• Hospital Pharmacy

• Retail Pharmacy

• Online Pharmacy

• Others

Market Breakup by Region

• North America

• Europe

• Asia Pacific

• Latin America

• Middle East and Africa

Global Acquired Orphan Blood Diseases Therapeutics Market Regional Analysis

North America is a significant regional market for the acquired orphan blood diseases segment, owing to the presence of an advanced healthcare infrastructure and services. There has been a significant surge in approvals of novel therapies by health regulatory bodies, which is poised to boost the acquired orphan blood disease therapeutics market size in the forecast period. In October 2023 , the United States Food and Drug Administration (FDA) approved Tibsovo (ivosidenib) developed by Servier Pharmaceuticals LLC (an international pharmaceutical company headquartered in France). The therapeutic drug can be used to treat refractory or relapsed myelodysplastic syndromes (MDS) with isocitrate dehydrogenase-1 (IDH1) mutation in adult patients. Tibsovo also has FDA Breakthrough Therapy designation and Orphan Drug designation to accelerate the drug development process for rare blood cancer treatment.

Europe holds a significant acquired orphan blood diseases therapeutics market share, which can be attributed to the presence of leading research institutes and biopharmaceutical companies propelling the growth of the market in the region. Moreover, the rising healthcare expenditure, increased research activities related to the development of acquired orphan blood disease drugs, and growing demand for effective treatment therapies for rare diseases are significantly accelerating the market growth.

Global Acquired Orphan Blood Diseases Therapeutics Market: Competitor Landscape

In February 2023, GC Biopharma, a biopharmaceutical company headquartered in South Korea, signed an agreement to purchase assets from Catalyst Biosciences, a clinical-stage biopharmaceutical company in the United States. The company plans to acquire the Catalyst Biosciences’ orphan hematology disease pipeline which includes 3 drug candidates. Marzeptacog alfa (MarzAA) is one such asset for the treatment of a rare bleeding disorder, undergoing a global phase 3 clinical trial. Further, the company intends to actively develop revolutionary treatment solutions for several orphan bleeding diseases. The growing incidence of acquisition and merger initiatives by pioneering biopharmaceutical companies are expected to elevate the acquired orphan blood diseases therapeutics growth in the forecast period.

The key features of the market report include patent analysis, grants analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:

• F. Hoffmann-La Roche Ltd.

• Pfizer, Inc.

• PTC Therapeutics

• AstraZeneca

• Novartis AG

• Takeda Pharmaceutical Company

• Bayer AG

• AbbVie Inc.

• Merck & Co. Inc.

• Bristol Myers Squibb

• Amgen Inc.

• Shire plc

• Alexion Pharmaceuticals Celgene Corporation

• Celgene Corporation

• Otsuka Holdings Co., Ltd.

• Novo Nordisk A/S

Kindly note that this only represents a partial list of companies, and the complete list has been provided in the report.

Key Highlights of the Report