Existing or abandoned pharmacotherapies can be developed for new use in other diseases through drug repositioning approaches. Finding new uses for known drugs is an area of growing interest. Compounds selected to be repositioned have often been through extensive testing such as toxicology, non-clinical safety, pharmacokinetic, and safety /tolerability assessments in healthy volunteers. This allows, investigators to build upon previous research and development efforts, accelerating and clearing the path towards clinical testing.
In summary key drug repositioning benefits are:
Shorter development time
Lower development costs
Higher success rates
As an illustration, over 2010-12, halfof the 46 FDA approvals in rare/orphan indications were repositioned drugs
As a private non-for-profit organisation, we strive to uncover the potential of existing molecules to address severe therapeutic unmet needs in rare diseases, thus giving better chances to existing drugs to reach these underserved patients.