Presentation at the B·Debate Conference on Drug Repurposing for Rare Diseases

H.M. Pharma Consultancy will present at the·B·Debate conference, Drug Repurposing for Rare Diseases. The Cure for the 21st Century, to be held in Barcelona on November 17 and 18, 2016. This event, jointly organized by Barcelona’s innovative health ecosystem, biocat and “la Caixa” Foundation, with Barcelona Science Park (PCB) and Hospital Sant Joan de Déu (HSJD), offers an interdisciplinary forum where scientists from the areas of chemistry, computation, biology and genomics, exchange ideas with clinical experts on rare diseases, and with representatives of entities involved in the development of therapies. Registration is available for 80 Euros.

Presentation details are as follows:

  • Presentation Type: Oral presentation
  • Title: Repurposed drugs for rare diseases – an attractive development option
  • Date and Time: Friday, November 18, 2016, 15:00 CEST
  • Presenter: Dr. Hermann Mucke


Developing known active ingredients for a rare disease for which no approved treatment is available can offer perspectives that are exciting, from both the medical and the commercial angle. The path from the laboratory to the patient can be quicker, and in any case the regulatory and exclusivity incentives are considerable. In principle, the synergy of these factors can overcompensate the drawbacks of the small market; however, drug repurposing for rare diseases also has some unique challenges which do not end with regulatory approval. Starting with recent examples of international patent applications claiming known drugs for new applications in rare neurological diseases, the possible development pathways and their timelines are outlined, and the associated problems are pointed out.