This is the question we asked ourselves as we brought together the speaking faculty and constructed the programme for the 4th Annual World Orphan Drug Congress in Geneva. As the orphan drug market grows at an enviable rate, the pharmaceutical industry have shifted focus and investments towards more niche disease indications, while biotech successfully commercialising their own pipelines are emerging as leading lights and highly attractive investment opportunities for the wider business community. As a result, we thought it interesting to understand where the next wave of orphan drugs may come from and which developers are driving forward future treatments to rare disease patients.
Utilising the invaluable resources on the European Commission Website and the highly respected portal for rare diseases and orphan drugs at Orphanet, we have compiled the Top 10 pharma and biotech companies with the largest number of orphan drug designations in their pipeline.
With over 7000 known rare diseases, and with the European Medicines Agency expecting 150+ orphan drug designations in 2013, the upward trend in orphan drug designations is a great sign for the future treatment of rare diseases. As an industry, we look forward to the translation of this research into treatments and cures. We hope you find the following slides interesting and please feel free to share it with your colleagues.