Orphan Drugs Market By Disease Type (Oncologic Diseases, Hematologic Others) By Indication (Non-Hodgkin Lymphoma, Cystic Fibrosis, Others) Regional Analysis & Forecast to 2026

  • OI-83
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  • Published date: Mar, 2019
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  • Life Sciences & Healthcare
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  • Pages

An orphan drug is nothing but a pharmaceutical agent, which has been developed to treat a specific rare medical condition, the condition itself being referred as “orphan disease”; which affects a small percentage of the population. Most of the rare diseases are genetic, and thus, are present throughout the person's life, even if symptoms do not immediately appear. Thus, compared to non-orphan drugs, these drugs do not guarantee feasible returns on investment. Despite, several government authorities are encouraging to develop and market such drugs. Cost for the development of these drugs is higher compared with non-orphan drugs cost. The orphan drugs are used in different indications including lymphoma, leukemia, cystic fibrosis, and others.

Market Overview

The market is anticipated to reach USD XX billion, by the end of 2022. A XX% CAGR will see prescription sales in the orphan sector grow at twice the rate of the overall prescription market, which is set to grow at just XX%, annually, between 2018 and 2026. Increasing the demand for advanced technological treatments and novel drug products to treat various rare diseases are provides robust market growth. The development of orphan drugs has been encouraged financially through US law via the Orphan Drug Act of 1983. The success of the original Orphan Drug Act in the USA is being adopted in other key markets, most prominently in Japan in 1993 and in the European Union in 2000.

Exclusive marketing in the United States for seven years and in Europe for ten years is permitted to orphan drug manufacturers. In the recent past, many companies have been entering this promising segment, as there is a provision for government incentives. The FDA has approved 80 new orphan indications in 2017 and 57 within just first eight months of 2018, the annually highest numbers since the passage of the Orphan Drug Act. In 2107, the FDA has granted orphan designations to over 429 unique drugs under development. Drug manufacturers are continuing to increase their focus on the development of therapies for orphan indications, and nearly half of the 42 new active substances (molecules not previously approved as a medicinal product) are launched in the United States in 2017 were orphan drugs.

The demand for the orphan drugs has raised notably due to a surge in the prevalence of rare diseases. Additionally, the rise in awareness among the people regarding rare diseases, drug development, and an increase in R&D investment drives the market growth. The impact of the driving factors is going to overcome the effects of restraints. Further, an increase in novel indications for known orphan drugs and increasing untapped economies are expected to account for new market opportunities to orphan drug manufacturers in the near future. On the other hand, the high cost of development of drugs is one of the major factors that is estimated to inhibit the growth of the overall orphan drugs market in the coming years.

Key Developments

  • Dec 2018, Stemline Therapeutics approved Elzonris (tagraxofusp) – the first drug licensed for rare cancer blastic plasmacytoid dendritic cell neoplasm (BPDCN) – and Servier’s Asparlas (calaspargase pegol), a long-acting drug for acute lymphoblastic leukaemia that also extends the dosing interval for patients.

  • Dec 2018, The FDA has approved Ultomiris (ravulizumab-cwvz) as an injection treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that leads to hemolysis.

  • Mar 2019, Mitochon Pharmaceuticals was awarded orphan drug designation for its mitochondrial targeted compound, MP-101developed for the treatment of Huntington’s disease.


The orphan drugs market is segmented on the basis of disease type, indication, and geography.

By Disease Type: Oncologic diseases, hematologic & immunologic diseases, metabolic diseases, neurologic diseases, infectious diseases, other rare diseases. Oncologic diseases occupied the dominant share. This is because of the array of different forms of rare cancers, including leukemia, myeloma, angiosarcoma, and others prevalent in the patient population.

By Indication: Non-Hodgkin Lymphoma, Cystic Fibrosis, Glioma, Pancreatic Cancer, Acute Myeloid Leukemia, Ovarian Cancer, Duchenne Muscular Dystrophy, Graft vs. Host Disease, Myeloma, Renal Cell Carcinoma, and Others

By Geographic Region: North America, Latin America, and Western Europe, Eastern Europe, and Asia pacific excluding japan, Middle East and Africa (MEA), Japan. North America dominating the global Orphan Drugs market followed by Europe. Asian Pacific region market is considered to rapidly evolving healthcare infrastructure and research and development, and this region is anticipating providing a robust growth of global Orphan Drugs market over the forecast period.

The key players involved in the orphan drugs market include Aegerion Pharmaceuticals, Inc., AbbVie Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche Ltd., GlaxoSmithKline plc, Celgene Corporation, Johnson & Johnson, Novartis AG, Pfizer Inc., and Sanofi

Report Description: The report covers in-depth analysis on Orphan Drugs Market. The report assesses the market products pipeline by stage of development (early development, pre-clinical, clinical and in approval), by application (lymphoma, leukemia, cystic fibrosis, and others). In addition, the report includes key insights on other development activities, including (but not limited to) – licensing (In and Out), collaborations, acquisitions, reimbursement, patent, and regulatory designations.

The report includes in-depth company profiles of key players in Orphan Drugs Market. The company profile includes key information on overview, financial highlights, product portfolio, business strategies, and key recent developments.

The report highlights information on emerging companies with potentially disruptive technologies and new market entrants.


  • Provides detailed analysis of the market structure along with forecast of the various segments and sub-segments of the Orphan Drugs Market.

  • Provides a comparative analysis of key marketed products and pipeline market products.

  • Provides key information on players involved on the Orphan Drugs Market.

  • Provides a complete overview of market segments and the regional outlook of Orphan Drugs Market.

  • Provides in-depth coverage of key news related toOrphan Drugs Market, including major mergers and acquisitions and product development updates such as clinical trial progression updates and regulatory updates

Our research works on a holistic 360° approach to deliver high quality, validated and reliable information in our market reports. The Market estimation and forecasting involve the following steps:

  • Data Collation (Primary & Secondary)

  • In-house Estimation (Based on proprietary databases and Models)

  • Market Triangulation

  • Forecasting

  • Market-related information is assembled from both primary and secondary sources. 

  • Primary sources involved participants from all global stakeholders such as experts from several related industries and suppliers that have been interviewed to obtain and verify critical information as well as to assess prospects of the market. The participants included are CXOs, VPs, and managers. Plus, our in-house industry experts having decades of industry experience contribute their consulting and advisory services. 

  • Secondary sources include public sources such as regulatory frameworks, government IT spending, government demographic indicators, industry association statistics, and company publications annual reports press releases along with paid sources such as Factiva, OneSource, Bloomberg among others. 

  • Top-down and bottom-up approaches: The overall market size was used in the top-down approach to estimate the sizes of other individual submarkets (mentioned in the market segmentation by product, type of manufacturing, and disease) through percentage splits from secondary and primary research. The bottom-up approach was also implemented (wherever applicable) for data extracted from secondary research to validate the market segment revenues obtained. 



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