Insight journal - Therapy

Orphan Diseases: recent partnering deal trends

Posted on 18 February 2014

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An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.

Orphan disease in the US is defined as that affecting fewer than 200,000 individuals. The term also reflects the industry’s neglect in developing treatments for orphan diseases and their focus to develop blockbuster drugs for common diseases. Pharma industry has not paid keen attention in the R&D of orphan drugs because it does not provide the necessary financial incentive to produce and market drugs to prevent or treat the disease.

Orphan diseases have become increasingly popular in the last decade. The reason for this rapid increase in dealmaking in this relatively unpopular therapy area could be due to collaboration among industries, venture capital investment, medical and technological breakthroughs that are changing the landscape of research in orphan diseases.

According to the Current Agreements life science deals and alliance database, there have been over 90 orphan diseases deals since 2009.

Figure 1: Orphan diseases partnering since 2009

orphan diseases 1







Source: Current Agreements, 2014

Unlike many other deal areas that involve top pharmaceutical (big pharma) companies taking part in deal making, orphan drugs has not been a popular area of deal making among the big pharma companies, with the exception of Pfizer and GSK. The reasons for this are pretty obvious in that big pharma prefer to seek out major therapeutic targets, in order to maximize return on R&D investment. However, this provides significant opportunity for smaller companies seeking a niche market opportunity.

Below is an insight into the most active orphan diseases dealmakers

Top orphan diseases dealmakers– 2009 to 2014

  • Edison Pharmaceuticals
  • FDA
  • GSK
  • National Institutes of Health
  • Pfizer
  • Viropharma
  • AGTC
  • Alitair Pharmacaeuticals
  • Aradigm
  • Cell Therapeutics

Source: Current Agreements, 2014

According to a recent article by a BBC Correspondent “The orphan drug market is currently said to be worth $50bn and is growing at about a rate of 6% per year”. The figure below illustrates the stage of development at which partnerships where announced for an orphan diseases drug target..

Figure 2: Orphan diseases partnering by stage of development since 2009

orphan diseases 2







Source: Current Agreements, 2014

As an emerging and exciting area for therapeutic intervention, orphan diseases deals can have a high headline deal value.

 Figure 3: Top orphan diseases deals by value since 2009

orphan disease 3














Source: Current Agreements, 2014

The Orphan Diseases Partnering Terms and Agreements report published by Current Partnering provides full details of over 90 orphan diseases deals announced since 2009 and includes average deal terms including headline, upfront, milestone and royalty rates. In addition, the report provides details of deals broken down by deal type, stage of development, company A-Z and technology type. More details here

There is a huge expectation in the advancement and development of drugs that demonstrate promise in diagnosis and treatment of orphan diseases. It would be interesting to witness the deal making activity and trends in this sector in the coming years.


The above information has been abstracted from the following resources:

Shamini Thiagarajan



Read: more orphan diseases deals in pharma, biotech, life science partnering deal news, insights and glossary

View: glossary definition for orphan drugs

Report: Orphan Diseases Partnering Terms and Agreements

View also: Other therapy type reports

View: Current Partnering’s Partnering Scorecard – view top life science partnering deals by value

View: Current Partnering’s Deal Metrics – the latest deal trend infographics for life science deal making

View: Current Partnering’s Big Pharma Deal Making Scorecard – latest trends in big pharma deal making activity

Signup: Current Partnering Dealmakers Update – weekly newsletter providing the latest life science industry deal news, deal making trends, partnering events – sign up now

Signup: Current Agreements Deals Review – monthly newsletter – reviewing the previous month’s life science deal making – partnering, M&A and financing – sign up now

Subscribe: Current Agreements life sciences partnering, M&A and financing deals database – find out more

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