Advancing treatments for rare diseases Together

About drug repositioning

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, half of the 46 FDA approvals in rare/orphan indications were repositioned drugs