What is currently the most popular rare disease being researched?

In Advocacy, Clinical Development by Matt Turner

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Is there such a thing as low hanging fruit when it comes to rare disease research?

While there are fewer than 300 approved orphan drugs for nearly 7000 rare diseases, it is interesting to analyse whether certain rare diseases receive greater attention than others.

Utilising the orphan drug designation list on the European Commission Website and the invaluable portal for rare diseases and orphan drugs at Orphanet, we have compiled the Top 7 most popular rare diseases currently being researched based on the number of orphan drug designations and therapies currently being developed for that particular disease. These will be released over the next 7 weeks within an eBook series that will highlight the companies and the development stage of the therapy.

As a little insight, below is the list of companies that have orphan drug designation for a therapy under development for Acute Myeloid Leukaemia:

  • AbbVie Ltd
  • Ambit Europe Limited
  • Aprea AB
  • Astex Therapeutics Limited
  • AstraZeneca AB
  • CanReg (Europe) Limited
  • Celator (UK) Ltd
  • Celgene Europe Limited
  • Cephalon Europe
  • Chroma Therapeutics Ltd
  • Cyclacel Limited
  • DC Prime BV
  • Diatos S. A.
  • Dr. Ulrich Granzer
  • EleosInc Limited
  • ERYtech Pharma S.A.
  • Fate Therapeutics, LTD
  • Galen limited
  • Genzyme Europe B.V.
  • Janssen-Cilag International NV
  • Kanisa Europe Limited, Mofo Notices Limited, C/Morrison & Foerster MNP
  • LTKFarma
  • Meda AB
  • Merck KGaA
  • Novartis Europharm Limited
  • Pfizer Limited
  • Sanofi-Aventis groupe
  • Sunesis Europe Ltd
  • Teva Pharma GmbH
  • Vion (UK) Limited, ℅ i3 Research
  • Xenetic Biosciences Plc
  • Clavis Pharma ASA

This increased competition in certain disease areas pose some potentially interesting questions. . How will multiple treatments for a single rare disease affect pricing levels? How will each developer demonstrate the value of its orphan drug against a rival treatment? And importantly, how can the policy makers and key opinion leaders incentivise the rare disease research community to explore possible therapies for continually neglected rare diseases?

Many of the leading orphan drug developers and key opinion leaders from the rare disease community are speaking, sponsoring and attending the 4th Annual World Orphan Drug Congress in Geneva. With unprecedented interest and endorsement already, he 2013 congress in Geneva is set to be the key strategic networking event for the orphan drug industry.

Click here to see the full agenda by downloading your copy of the brochure.

Limited delegate places are available so book now to guarantee your seat at the table.