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Acquired Orphan Blood Diseases Therapeutics Market Size, Share & Industry Analysis, 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) By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Others) Others and Regional Forecast, 2023-2030

Region : Global | Report ID: FBI102010 | Status : Ongoing



Acquired orphan blood disease is a type of rare blood disorder, which occurs due to an insufficient amount of red cells in the blood. The disease is characterized by the inability to produce red blood cells. Moreover, improper functioning of bone marrow also results in the lack of red blood cells in the blood, which in turn results in a decrease in platelet numbers. Some of the common types of orphan disease which results in acquired orphan blood disease are paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome, acquired agranulocytosis, and polycythemia vera (PV). Rising prevalence of blood-related disorders, growing awareness among people about these disorders, and improved healthcare infrastructure are some of the factors that are expected to drive the market growth during the forecast period.

Key Market Driver -

Rising prevalence of blood-related disorders

Key Market Restraint -

Stringent regulatory policies

Rising prevalence of blood-related disorders, growing geriatric population, increasing awareness among people about healthcare, and government initiatives for increasing awareness regarding occurrences of blood-realted disorders are some of the major factors that are driving the market growth.

However, long duration of time required for the approval of orphan blood diseases drug and stringent governemnt regulations  are some of the factors expected to restrain the growth of acquired orphan blood diseases therapeutics market during the forecast period.

Market Segmentation:

By therapy, the market can be segmented into recombinant factor, immunoglobulin infusion therapy, activated prothrombin complex concentrate, thrombopoietin receptor agonists, and others. By disease indication, the market can be segmented into acquired agranulocytosis, acquired hemophilia, acquired von willebrand syndrome, paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome, and others. By distribution channel, the market can be segmented into hospital pharmacy, retail pharmacy, and others.

Geographically, the acquired orphan blood diseases therapeutics market can be segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa.

Key Players Covered:

The major companies in the global acquired orphan blood diseases therapeutics market report includes Amgen Inc., Shire plc, Alexion Pharmaceuticals, Celgene Corporation, Rigel Pharmaceuticals, Inc., Otsuka Pharmaceutical Pvt. Ltd., Novo Nordisk Inc. and other prominent players.

 Key Insights

  • Reimbursement scenario - For Key Countries

  • Recent Industry Developments – Partnerships, Mergers & Acquisitions

  • Prevalence Data of Acquired Orphan Blood Diseases (For Diseases under focus), for Key Countries

  • Pipeline Analysis

Regional Analysis:

The global acquired orphan blood diseases therapeutics market is expected to be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is anticipated to lead the global acquired orphan blood diseases therapeutics market due to an increasing prevalence of blood-related disorders and increasing research and development investment in the U.S. According to the Statista, about 18,000 patients had haemophilia and over 13,000 patients had von willebrand syndrome, as of 2018.

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Europe is likely to have substantial market growth throughout the forecast period owing to the rising prevalence of blood-related disorders. Asia Pacific and Latin America are expected to register a fast market growth during the forecast period due to the increasing awareness about the acquired orphan blood diseases and its treatment among the people. On the other hand, Middle East and Africa is projected to have comparatively less CAGR in the acquired orphan blood diseases therapeutics market in the upcoming years.




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

  • Other

By Distribution Channel

  • Hospital Pharmacy

  • Retail Pharmacy

  • Others

By Geography

  • North America (USA, Canada)

  • Europe (UK, Germany, France, Italy, Spain, Scandinavia and Rest of Europe)

  • Asia Pacific (Japan, China, India, Australia, Southeast Asia and Rest of Asia Pacific)

  • Latin America (Brazil, Mexico, Rest of Latin America)

  • Middle East & Africa (South Africa, GCC and Rest of Middle East & Africa)

Acquired Orphan Blood Diseases Therapeutics Industry Developments

  • In February 2019, U.S FDA approved Cablivi (caplacizumab-yhdp) injection, the first therapy indicated, in combination with plasma exchange and immunosuppressive therapy for the treatment of thrombotic thrombocytopenic purpura (aTTP).

  • In March 2018, ADDMEDICA received U.S. FDA approval for Orphan Drug Siklos, first and sole hydroxyurea-based treatment for paediatric patients with sickle cell anaemia.

  • Ongoing
  • 2022
  • 2019-2021


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