Global Orphan Drugs Market Report to 2030 – Players Include Amgen, Bristol-Myers Squibb and Pfizer –

Global Orphan Drugs Market Report to 2030 – Players Include Amgen, Bristol-Myers Squibb and Pfizer –

Global Orphan Drugs Market Report to 2030 – Players Include Amgen, Bristol-Myers Squibb and Pfizer –

DUBLIN–(BUSINESS WIRE)–The “Orphan Drugs Market by Disease Type: Global Opportunity Analysis and Industry Forecast, 2021-2030” report has been added to’s offering.

The global orphan drugs market was valued at $1,40,000.0 million in 2020 and is projected to reach $4,35,686.3 million by 2030 registering a CAGR of 11.8% from 2021 to 2030.

The pharmaceutical agents administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs. Orphan drugs are used to treat rare (orphaned) diseases prevalent in a very small patient pool. Thus, the pharmaceutical industry takes little interest in their development. Moreover, their development requires high cost and the chances of returns on investment are comparatively less when compared to non-orphan drugs. These drugs treat different rare diseases, such as non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, and Duchenne muscular dystrophy, graft vs. host disease, and others.

The main factor that drives the growth of the orphan drugs market includes increase in prevalence of rare diseases. In addition, favorable government policies, and availability of market exclusivity for orphan drugs developers further supplement the global orphan drugs market growth.

Conversely, limited patient pool for clinical trials and product marketing, and high treatment costs per patient are expected to obstruct the growth of the market during the forecast years. On the other hand, high growth potential in untapped emerging economies, and rise in novel indications for known orphan drugs are expected to offer profitable opportunities for the growth of the market during the forecast period. The global orphan drugs market is segmented on the basis of disease type, and region to provide a detailed assessment of the market.

By disease type, it is categorized into oncological diseases, metabolic diseases, hematologic & immunologic diseases, infectious diseases, neurological diseases, and other rare diseases such as transplantation diseases, gastrointestinal diseases, dermatologic diseases, perinatal & congenital abnormalities, and others. Oncological diseases segment is further divided into acute myeloid leukemia (AML), pancreatic cancer, ovarian cancer, multiple myeloma, renal cell carcinoma, and others.

In addition, the metabolic diseases segment is again classified into hunter syndrome, Fabry disease, Gaucher disease, hypoparathyroidism, and others. Furthermore, hematologic & immunologic diseases segment is segmented into hereditary angioedema (HAE), hemophilia, and others. Moreover, neurological diseases segment is further segmented into Duchenne muscular dystrophy (DMD), Alzheimer’s disease, Huntington’s disease, and others. Region wise, the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

Key Benefits

  • The study provides an in-depth analysis of the global orphan drugs market along with the current trends and future estimations to elucidate the imminent investment pockets
  • A comprehensive analysis of the factors that drive and restrict the market growth is provided in the report
  • Comprehensive quantitative analysis of the industry from 2020 to 2030 is provided to enable the stakeholders to capitalize on the prevailing market opportunities
  • Extensive analysis of the key segments of the industry helps to understand the application and products of orphan drugs used across the globe
  • Key market players and their strategies have been analyzed to understand the competitive outlook of the market

Market Dynamics


  • Increase in Prevalence of Rare Diseases
  • Favorable Government Policies
  • Availability of Exclusivity for Orphan Drugs


  • Limited Patient Pool for Clinical Trials and Product Marketing
  • High Treatment Costs Per Patient


  • High Growth Potential in Untapped Emerging Economies
  • Use of Orphan Drugs for Novel Conditions/Indications

Key Segments

By Disease Type

  • Oncological Diseases
  • Acute Myeloid Leukemia (AML)
  • Pancreatic Cancer
  • Ovarian cancer
  • Multiple Myeloma
  • Renal Cell Carcinoma
  • Others
  • Metabolic Diseases
  • Hunter Syndrome
  • Fabry Disease
  • Gaucher Disease
  • Hypoparathyroidism
  • Others
  • Hematologic & Immunologic Diseases
  • Hereditary Angioedema (HAE)
  • Hemophilia
  • Others
  • Infectious Diseases
  • Neurologic Diseases
  • Duchenne Muscular Dystrophy
  • Alzheimer’s Disease
  • Huntington’s Disease
  • Others
  • Other Rare Diseases

By Region

  • North America
  • US
  • Canada
  • Mexico
  • Europe
  • Germany
  • France
  • UK
  • Italy
  • Spain
  • Rest of Europe
  • Asia-Pacific
  • Japan
  • China
  • Australia
  • India
  • Rest of Asia-Pacific
  • Brazil
  • Turkey
  • South Africa
  • Rest of LAMEA

Key Players Profiled

  • Abbvie Inc
  • Amgen Inc
  • Amryt Pharma Plc
  • Bristol-Myers Squibb Company
  • F Hoffmann-La Roche AG
  • GlaxoSmithkline PLC
  • Johnson & Johnson (Janssen Global Services, LLC)
  • Novartis AG (Sandoz)
  • Pfizer Inc
  • Sanofi SA

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