Advancing treatments for rare diseases Together

About drug repositioning

Drug repositioning is the application of known drugs (existing on the market or abandoned) to treat new diseases. 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, 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 treating patients.

In summary key drug repositioning benefits are (compared to classical drug development):

  • 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.