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Global Acquired Orphan Blood Diseases Therapeutics Market: By Therapy: Recombinant Factor, Immunoglobulin Infusion Therapy, Thrombopoietin Receptor Agonists, Activated Prothrombin Complex Concentrate, Others; By Indication; By Distribution Channel; Regional Analysis; Supplier Landscape; 2024-2032
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 2024-2032, 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.
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
| REPORT FEATURES | DETAILS |
| Base Year | 2023 |
| Historical Period | 2017-2023 |
| Forecast Period | 2024-2032 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Therapy |
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| Breakup by Indication |
|
| Breakup by Distribution Channel |
|
| Breakup by Region |
|
| Market Dynamics |
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| Competitive Landscape |
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| Companies Covered |
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*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
1.4 Research Methodology
2 Executive Summary
3 Global Acquired Orphan Blood Diseases Therapeutics Market Overview
3.1 Global Acquired Orphan Blood Diseases Therapeutics Market Historical Value (2017-2023)
3.2 Global Acquired Orphan Blood Diseases Therapeutics Market Forecast Value (2024-2032)
4 Global Acquired Orphan Blood Diseases Therapeutics Market Landscape*
4.1 Global Acquired Orphan Blood Diseases Therapeutics: Developers Landscape
4.1.1 Analysis by Year of Establishment
4.1.2 Analysis by Company Size
4.1.3 Analysis by Region
4.2 Global Acquired Orphan Blood Diseases Therapeutics: Product Landscape
4.2.1 Analysis by Therapy
4.2.2 Analysis by Indication
5 Global Acquired Orphan Blood Diseases Therapeutics Market Dynamics
5.1 Market Drivers and Constraints
5.2 SWOT Analysis
5.2.1 Strengths
5.2.2 Weaknesses
5.2.3 Opportunities
5.2.4 Threats
5.3 Porter’s Five Forces Model
5.3.1 Bargaining Power of Suppliers
5.3.2 Bargaining Power of Buyers
5.3.3 Threat of New Entrants
5.3.4 Threat of Substitutes
5.3.5 Degree of Rivalry
5.4 Key Demand Indicators
5.5 Key Price Indicators
5.6 Industry Events, Initiatives, and Trends
5.7 Value Chain Analysis
6 Global Acquired Orphan Blood Diseases Therapeutics Market Segmentation (2017-2032)
6.1 Global Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Therapy
6.1.1 Market Overview
6.1.2 Recombinant Factor
6.1.3 Immunoglobulin Infusion Therapy
6.1.4 Thrombopoietin Receptor Agonists
6.1.5 Activated Prothrombin Complex Concentrate
6.1.6 Others
6.2 Global Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Indication
6.2.1 Market Overview
6.2.2 Acquired Agranulocytosis
6.2.3 Acquired Hemophilia
6.2.4 Acquired Von Willebrand Syndrome
6.2.5 Paroxysmal Nocturnal Hemoglobinuria (PNH)
6.2.6 Myelodysplastic Syndrome
6.2.7 Others
6.3 Global Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Distribution Channel
6.3.1 Market Overview
6.3.2 Hospital Pharmacy
6.3.3 Retail Pharmacy
6.3.4 Online Pharmacy
6.3.5 Others
6.4 Global Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Region
6.4.1 Market Overview
6.4.2 North America
6.4.3 Europe
6.4.4 Asia Pacific
6.4.5 Latin America
6.4.6 Middle East and Africa
7 North America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032)
7.1 North America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Therapy
7.1.1 Market Overview
7.1.2 Recombinant Factor
7.1.3 Immunoglobulin Infusion Therapy
7.1.4 Thrombopoietin Receptor Agonists
7.1.5 Activated Prothrombin Complex Concentrate
7.1.6 Others
7.2 North America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Indication
7.2.1 Market Overview
7.2.2 Acquired Agranulocytosis
7.2.3 Acquired Hemophilia
7.2.4 Acquired Von Willebrand Syndrome
7.2.5 Paroxysmal Nocturnal Hemoglobinuria (PNH)
7.2.6 Myelodysplastic Syndrome
7.2.7 Others
7.3 North America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Country
7.3.1 United States of America
7.3.2 Canada
8 Europe Acquired Orphan Blood Diseases Therapeutics Market (2017-2032)
8.1 Europe Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Therapy
8.1.1 Market Overview
8.1.2 Recombinant Factor
8.1.3 Immunoglobulin Infusion Therapy
8.1.4 Thrombopoietin Receptor Agonists
8.1.5 Activated Prothrombin Complex Concentrate
8.1.6 Others
8.2 Europe Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Indication
8.2.1 Market Overview
8.2.2 Acquired Agranulocytosis
8.2.3 Acquired Hemophilia
8.2.4 Acquired Von Willebrand Syndrome
8.2.5 Paroxysmal Nocturnal Hemoglobinuria (PNH)
8.2.6 Myelodysplastic Syndrome
8.2.7 Others
8.3 Europe Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Country
8.3.1 United Kingdom
8.3.2 Germany
8.3.3 France
8.3.4 Italy
8.3.5 Others
9 Asia Pacific Acquired Orphan Blood Diseases Therapeutics Market (2017-2032)
9.1 Asia Pacific Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Therapy
9.1.1 Market Overview
9.1.2 Recombinant Factor
9.1.3 Immunoglobulin Infusion Therapy
9.1.4 Thrombopoietin Receptor Agonists
9.1.5 Activated Prothrombin Complex Concentrate
9.1.6 Others
9.2 Asia Pacific Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Indication
9.2.1 Market Overview
9.2.2 Acquired Agranulocytosis
9.2.3 Acquired Hemophilia
9.2.4 Acquired Von Willebrand Syndrome
9.2.5 Paroxysmal Nocturnal Hemoglobinuria (PNH)
9.2.6 Myelodysplastic Syndrome
9.2.7 Others
9.3 Asia Pacific Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Country
9.3.1 China
9.3.2 Japan
9.3.3 India
9.3.4 ASEAN
9.3.5 Australia
9.3.6 Others
10 Latin America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032)
10.1 Latin America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Therapy
10.1.1 Market Overview
10.1.2 Recombinant Factor
10.1.3 Immunoglobulin Infusion Therapy
10.1.4 Thrombopoietin Receptor Agonists
10.1.5 Activated Prothrombin Complex Concentrate
10.1.6 Others
10.2 Latin America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Indication
10.2.1 Market Overview
10.2.2 Acquired Agranulocytosis
10.2.3 Acquired Hemophilia
10.2.4 Acquired Von Willebrand Syndrome
10.2.5 Paroxysmal Nocturnal Hemoglobinuria (PNH)
10.2.6 Myelodysplastic Syndrome
10.2.7 Others
10.3 Latin America Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Country
10.3.1 Brazil
10.3.2 Argentina
10.3.3 Mexico
10.3.4 Others
11 Middle East and Africa Acquired Orphan Blood Diseases Therapeutics Market (2017-2032)
11.1 Middle East and Africa Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Therapy
11.1.1 Market Overview
11.1.2 Recombinant Factor
11.1.3 Immunoglobulin Infusion Therapy
11.1.4 Thrombopoietin Receptor Agonists
11.1.5 Activated Prothrombin Complex Concentrate
11.1.6 Others
11.2 Middle East and Africa Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Indication
11.2.1 Market Overview
11.2.2 Acquired Agranulocytosis
11.2.3 Acquired Hemophilia
11.2.4 Acquired Von Willebrand Syndrome
11.2.5 Paroxysmal Nocturnal Hemoglobinuria (PNH)
11.2.6 Myelodysplastic Syndrome
11.2.7 Others
11.3 Middle East and Africa Acquired Orphan Blood Diseases Therapeutics Market (2017-2032) by Country
11.3.1 Saudi Arabia
11.3.2 United Arab Emirates
11.3.3 Nigeria
11.3.4 South Africa
11.3.5 Others
12 Regulatory Framework
12.1 Regulatory Overview
12.1.1 US FDA
12.1.2 EU EMA
12.1.3 INDIA CDSCO
12.1.4 JAPAN PMDA
12.1.5 Others
13 Patent Analysis
13.1 Analysis by Type of Patent
13.2 Analysis by Publication year
13.3 Analysis by Issuing Authority
13.4 Analysis by Patent Age
13.5 Analysis by CPC Analysis
13.6 Analysis by Patent Valuation
13.7 Analysis by Key Players
14 Grants Analysis
14.1 Analysis by Year
14.2 Analysis by Amount Awarded
14.3 Analysis by Issuing Authority
14.4 Analysis by Grant Application
14.5 Analysis by Funding Institute
14.6 Analysis by Departments
14.7 Analysis by Recipient Organization
15 Funding and Investment Analysis
15.1 Analysis by Funding Instances
15.2 Analysis by Type of Funding
15.3 Analysis by Funding Amount
15.4 Analysis by Leading Players
15.5 Analysis by Leading Investors
15.6 Analysis by Geography
16 Partnership and Collaborations Analysis
16.1 Analysis by Partnership Instances
16.2 Analysis by Type of Partnership
16.3 Analysis by Leading Players
16.4 Analysis by Geography
17 Supplier Landscape
17.1 F. Hoffmann-La Roche Ltd.
17.1.1 Financial Analysis
17.1.2 Product Portfolio
17.1.3 Demographic Reach and Achievements
17.1.4 Mergers and Acquisitions
17.1.5 Certifications
17.2 Pfizer, Inc.
17.2.1 Financial Analysis
17.2.2 Product Portfolio
17.2.3 Demographic Reach and Achievements
17.2.4 Mergers and Acquisitions
17.2.5 Certifications
17.3 PTC Therapeutics
17.3.1 Financial Analysis
17.3.2 Product Portfolio
17.3.3 Demographic Reach and Achievements
17.3.4 Mergers and Acquisitions
17.3.5 Certifications
17.4 AstraZeneca
17.4.1 Financial Analysis
17.4.2 Product Portfolio
17.4.3 Demographic Reach and Achievements
17.4.4 Mergers and Acquisitions
17.4.5 Certifications
17.5 Novartis AG
17.5.1 Financial Analysis
17.5.2 Product Portfolio
17.5.3 Demographic Reach and Achievements
17.5.4 Mergers and Acquisitions
17.5.5 Certifications
17.6 Takeda Pharmaceutical Company
17.6.1 Financial Analysis
17.6.2 Product Portfolio
17.6.3 Demographic Reach and Achievements
17.6.4 Mergers and Acquisitions
17.6.5 Certifications
17.7 Bayer AG
17.7.1 Financial Analysis
17.7.2 Product Portfolio
17.7.3 Demographic Reach and Achievements
17.7.4 Mergers and Acquisitions
17.7.5 Certifications
17.8 AbbVie Inc.
17.8.1 Financial Analysis
17.8.2 Product Portfolio
17.8.3 Demographic Reach and Achievements
17.8.4 Mergers and Acquisitions
17.8.5 Certifications
17.9 Merck & Co. Inc.
17.9.1 Financial Analysis
17.9.2 Product Portfolio
17.9.3 Demographic Reach and Achievements
17.9.4 Mergers and Acquisitions
17.9.5 Certifications
17.10 Bristol Myers Squibb
17.10.1 Financial Analysis
17.10.2 Product Portfolio
17.10.3 Demographic Reach and Achievements
17.10.4 Mergers and Acquisitions
17.10.5 Certifications
17.11 Amgen Inc.
17.11.1 Financial Analysis
17.11.2 Product Portfolio
17.11.3 Demographic Reach and Achievements
17.11.4 Mergers and Acquisitions
17.11.5 Certifications
17.12 Shire plc
17.12.1 Financial Analysis
17.12.2 Product Portfolio
17.12.3 Demographic Reach and Achievements
17.12.4 Mergers and Acquisitions
17.12.5 Certifications
17.13 Alexion Pharmaceuticals Celgene Corporation,
17.13.1 Financial Analysis
17.13.2 Product Portfolio
17.13.3 Demographic Reach and Achievements
17.13.4 Mergers and Acquisitions
17.13.5 Certifications
17.14 Celgene Corporation
17.14.1 Financial Analysis
17.14.2 Product Portfolio
17.14.3 Demographic Reach and Achievements
17.14.4 Mergers and Acquisitions
17.14.5 Certifications
17.15 Otsuka Holdings Co., Ltd.
17.15.1 Financial Analysis
17.15.2 Product Portfolio
17.15.3 Demographic Reach and Achievements
17.15.4 Mergers and Acquisitions
17.15.5 Certifications
17.16 Novo Nordisk A/S
17.16.1 Financial Analysis
17.16.2 Product Portfolio
17.16.3 Demographic Reach and Achievements
17.16.4 Mergers and Acquisitions
17.16.5 Certifications
List not exhaustive
18 Global Acquired Orphan Blood Diseases Therapeutics Market – Distribution Model (Additional Insight)
18.1 Overview
18.2 Potential Distributors
18.3 Key Parameters for Distribution Partner Assessment
19 Key Opinion Leaders (KOL) Insights (Additional Insight)
20 Company Competitiveness Analysis (Additional Insight)
20.1 Very Small Companies
20.2 Small Companies
20.3 Mid-Sized Companies
20.4 Large Companies
20.5 Very Large Companies
21 Payment Methods (Additional Insight)
21.1 Government Funded
21.2 Private Insurance
21.3 Out-of-Pocket
*Additional insights provided are customisable as per client requirements.
* The coverage of the Market Landscape section depends on the data availability and may cover a minimum of 80% of the total market. The EMR team strives to make this section as comprehensive as possible.
Key Questions Answered in the Report
The market is anticipated to grow at a CAGR of 7.5% during the forecast period of 2024-2032, driven by the emergence of novel therapies and the rising expenditure on the healthcare sector.
Advancements in the healthcare sector and robust support from the government, are fuelling the demand for acquired orphan blood diseases therapeutics.
One of the significant trends witnessed in the market 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 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 who are dependent on blood transfusions and dealing with anemia despite anti-C5 treatments.
Based on the therapy, the market is segmented into recombinant factor, immunoglobulin infusion therapy, thrombopoietin receptor agonists, and activated prothrombin complex concentrate, among others.
Based on the indication, the market is segmented into acquired agranulocytosis, acquired hemophilia, acquired Von Willebrand syndrome, paroxysmal nocturnal hemoglobinuria (PNH), and myelodysplastic syndrome, among others.
Major distribution channels include hospital pharmacy, retail pharmacy, and online pharmacy, among others.
The major regions of the market include North America, Europe, Asia Pacific, Latin America, Middle East, and Africa.
Key players involved in the market are 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., and Novo Nordisk A/S.
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