Orphan Drug Market Overview

Orphan Drug Market are drugs developed to treat rare diseases that only a small proportion of people have and often have limited treatment options. The main drivers of the development of orphan pharmaceuticals are laws such as the US's Orphan Drug Act (ODA), which provides incentives to companies that find treatments for rare diseases. However, tiny patient populations, hefty development costs, and complicated regulatory criteria make it difficult to produce orphan medications. Notwithstanding these obstacles, the market for orphan drugs is expanding quickly due to rising awareness of rare diseases, developments in customized treatment, and the global expansion of legislative incentives.

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Dynamics of the Orphan Drug Market

The market for orphan drugs is growing faster because to emerging technologies.

Technologies like gene editing, artificial intelligence, and sophisticated drug delivery systems are driving the orphan drug business, giving it a significant influence. Innovations in illness treatment, including gene therapy, antibody therapy, enzyme replacement therapy, small molecule drugs, and drug repositioning, are expanding the range of available treatments. For example, the rare genetic condition cystic fibrosis, which causes respiratory failure and recurrent lung infections, is caused by mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene. existing drugs that use AI algorithms to change CFTR activity, creating a more efficient path for drug development.

Orphan Drug Market Regional Insights

In 2023, North America held the largest share of the orphan medicine market. The region is anticipated to maintain its dominance by 2023 and increase at a compound annual growth rate (CAGR) of 6.5% for a few years. In North America, the United States holds the largest market share. The FDA-approved orphan medicine's seven-year advertising exclusivity, tax deductions, and reduced user fees after approval for a specific indication are some of the elements driving the market's expansion in the United States.

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Orphan Drug Market Segmentation

by Product

Biological
Non-Biological

 

In terms of product type, the biologicals segment accounted for the greatest proportion of the orphan drug market in 2023 and is projected to increase at a compound annual growth rate (CAGR) of 9.6% through 2030. The market for rare disease treatment is expanding because to biologicals. These medications provide specialized treatments for diseases such uncommon malignancies and genetic abnormalities. More complicated and effective biological treatments have been made possible by biotechnology advancements, and their development has been aided by regulatory incentives. Biologics are becoming increasingly important in this industry as patients' requirements are being met by the rising prevalence and awareness of uncommon diseases.

by Disease Type

Oncology
Pancreatic Cancer
Ovarian cancer
Multiple Myeloma
Renal Cell Carcinoma
Others
Hematology
Hereditary Angioedema (HAE)
Hemophilia
Others
Neurology
Duchenne Muscular Dystrophy
Alzheimer's Disease
Huntington's Disease
Others
Cardiovascular

by Therapy Type

Oncology
Neuromuscular
Respiratory
Hematology
Others

by Distribution Channel

Hospital pharmacies
Retail pharmacies
Online pharmacies
others

Orphan Drug Market Key Players

1. Takeda Pharmaceutical Company Limited
2. Novartis AG
3. Johnson & Johnson
4. F. Hoffmann-La Roche Ltd
5. Bristol-Myers Squibb Company (Celgene Corporation)
6. Amgen Inc.
7. Bayer AG
8. Alexion Pharmaceutical Inc.
9. Novo Nordisk A/S
10. Pfizer Inc.
11. Eli Lilly and Company
12. Merck KGaA
13. Bristol-Myers Squibb Company
14. Amgen Inc.,
15. Alexion Pharmaceutical Inc
16. Bayer AG
17. Bristol Myers Squibb Co
18. Celegne Corporation
19. DAIICHI SANKYO Company Ltd
20. F. Hoffmann La-Roche
21. GlaxoSmithKline PLC
22. Sawai Pharmaceutical (Japan)
23. LG Chem (South Korea)
24. Chia Tai Tianqing Pharmaceutical Group (China)
25. Cipla (India)
26. Dr. Reddy's Laboratories (India)
27. Sun Pharmaceutical Industries (India)

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Key questions answered in the Orphan Drug Market are:

  • What is Orphan Drug?
  • What will be the CAGR at which the Orphan Drug market will grow?
  • What is the growth rate of the Orphan Drug Market?
  • What are the industry trends that can be implemented to generate additional revenue streams for the Orphan Drug Market?
  • Who are the leading companies and what are their portfolios in Orphan Drug Market?
  • What segments are covered in the Orphan Drug Market?
  • Which are the factors expected to drive the Orphan Drug market growth?

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