Rare- & orphan disease drug development
This paper describes the orphan disease pipeline of Swedish companies and presents statistics over orphan drug designations granted to Swedish companies by the US Food and Drug Administration (FDA) and the European Medicines Agency.
- Swedish companies have been granted 60 orphan designations in the EU and 47 in the US from January 2000 until December 2018
- Oncology is the leading orphan drug therapy area accounting for about 38% of the total Swedish pipeline
- Neurology, immunology/rheumatology and transplantation are the next leading therapy areas, accounting for another 26% of the pipeline
- Sobi is the largest Swedish company active in this area with several approved orphan drugs
- Perspectives on orphan drug development is given by HealthCap, Immedica Pharma, Karolinska Institutet, NDA Group, and Sobi
- A list of Swedish orphan disease pipeline projects is outlined