Global Orphan Drug Clinical Pipeline Insight 2022

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“Global Orphan Drug Clinical Pipeline Insight 2022” report gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.


As of 2016, almost 30 million people in the U.S. and 350 million people worldwide suffer from rare ailments known as “Orphan Diseases.” The name comes from the treatment meted out for such diseases, wherein the pharma industry was reluctant to develop drugs for such illnesses due to their economic unviability.


Elaborating the concept, a rare disease or orphan diseases are the one’s which affects only a small population. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. Consequently, in earlier times such truncated prevailed diseases were least attractive to the researchers and the investors. Previously, the market for orphan drugs was not as lucrative as it is today.


Nevertheless, the Scenario has changed after the Orphan Drug Act (ODA), 1983 was passed in United States and later various countries enacted similar laws. The number of requests for orphan drug designation received by FDA’s Office of Orphan Products Development (OOPD) has grown dramatically in recent years and is prompting FDA to adjust its timeframes for reviewing orphan drug designations in order to meet the demand.


The Orphan drug Market has seen a tremendous growth in last 2 decades. There has been a paradigm shift in the pharma or biopharmaceutical market with focus on research and development for unmet medical / clinical needs, investing resources in developing drugs to treat rare clinical conditions thus targeting small pool of patients. More than 7000 diseases have been given the designation of being rare. Subsequently, the global market is ever growing for the orphan drugs due to many factors including tax credits, grants, waived FDA fees, reduced timelines for clinical development and a higher probability of regulatory approval. Other aspects such as the patent expirations, generic completions and drying pipelines are also enhancing the market growth.


Orphan drugs are not merely lifesavers for patients suffering from these debilitating diseases, but also a huge growth opportunity for the pharma industry. With ROIs nearly twice that of non-orphan drugs, R&D activity for newer orphan drugs is expected to witness an exponential increase in the near future. Most big pharma players are capitalizing on rare disease treatments by enhancing their pipelines or by acquiring promising molecules of smaller companies. While the orphan drug market is mostly restricted to the U.S. and Europe now, pharma companies are likely to start tapping into the Asian market which has a high population of untreated rare diseases. It seems growth of the global orphan drug market is inevitable.


“Global Orphan Drug Clinical Pipeline Insight 2022” Report Highlight

1. Global Orphan Drug Market Overview

  1.1 Market Overview

  1.2 Global Orphan Drug Clinical Pipeline Overview


2. Global Orphan Drug Designation Criteria

  2.1 US Food and Drug Administration (US FDA)

  2.2 European Medicines Agency (EMA)

  2.3 Japan

  2.4 Taiwan

  2.5 Korean Food and Drug Administration (KFDA)

  2.6 Australia


Global Orphan Drug Clinical Pipeline by Company, Indication & Phase


3. Orphan Drug Development Phase: Unknown

  3.1 Overview

  3.2 Clinical Pipeline Insight


4. Orphan Drug Development Phase: Research

  4.1 Overview

  4.2 Clinical Pipeline Insight


5. Orphan Drug Development Phase: Preclinical

  5.1 Overview

  5.2 Clinical Pipeline Insight


6. Orphan Drug Development Phase: Clinical

  6.1 Overview

  6.2 Clinical Pipeline Insight


7. Orphan Drug Development Phase: Phase-0

  7.1 Overview

  7.2 Clinical Pipeline Insight


8. Orphan Drug Development Phase: Phase - I

  8.1 Overview

  8.2 Clinical Pipeline Insight


9. Orphan Drug Development Phase: Phase - I/II

  9.1 Overview

  9.2 Clinical Pipeline Insight


10. Orphan Drug Development Phase: Phase - II

  10.1 Overview

  10.2 Clinical Pipeline Insight


11. Orphan Drug Development Phase: Phase - II/III

  11.1 Overview

  11.2 Clinical Pipeline Insight


12. Orphan Drug Development Phase: Phase - III

  12.1 Overview

  12.2 Clinical Pipeline Insight


13. Orphan Drug Development Phase: Preregistration

  13.1 Overview

  13.2 Clinical Pipeline Insight


14. Orphan Drug Development Phase: Registered

  14.1 Overview

  14.2 Clinical Pipeline Insight


15. Marketed Orphan Drug Clinical Insight by Company, Indication & Phase

  15.1 Overview

  15.2 Clinical Pipeline Insight


16. Discontinued & Suspended Orphan Drugs in Clinical Pipeline by Company, Indication & Phase

  16.1 No Development Reported

  16.2 Discontinued

  16.3 Preregistration Submission Withdrawal

  16.4 Marketed Withdrawal

  16.5 Suspended


17. Competitive Landscape

  17.1 AOP Orphan

  17.2 Agenus

  17.3 Alexion

  17.4 Bristol Myers Squibb

  17.5 Biogen Idec

  17.6 Celgene

  17.7 Eli Lilly

  17.8 Genethon

  17.9 Genzyme Corporation

  17.10 Glaxosmithkline

  17.11 Merck

  17.12 Novartis Pharmaceuticals

  17.13 Orphan Europe

  17.14 Pfizer

  17.15 Prosensa

  17.16 Rare Disease Therapeutics

  17.17 Roche

  17.18 Sanofi

  17.19 Shire

  17.20 Teva Pharmaceutical

Figure 1 1: Global Orphan Drugs Market Value (US$ Billion), 2015-2022

Figure 1 2: US - Orphan Drugs Market Value (US$ Billion), 2015-2022

Figure 1 3: Europe - Orphan Drugs Market Value (US$ Billion), 2015-2022

Figure 1 4: Asia* - Orphan Drugs Market Value (US$ Billion), 2015-2022

Figure 1 5: Global - Orphan Drug Clinical Pipeline by Phase (%), 2016 till 2022

Figure 1 6: Global - Orphan Drug Clinical Pipeline by Phase (Numbers), 2016 till 2022

Figure 1 7: Global - Orphan Drug Clinical Pipeline by Phase (%),2016 till 2022

Figure 1 8: Global - Orphan Drug Clinical Pipeline by Phase (Numbers), 2016 till 2022

Figure 17 1: AOP Orphan

Figure 17 2: Agenus Pipeline

Figure 17 3: Genethon Drug Pipeline

Figure 17 4: Genzyme Drug Pipeline

Figure 17 5: Orphan Europe Pipeline

Figure 17 6: Prosensa Drug Pipeline

Figure 17 7: Shire Drug Pipeline