Drug repositioning for rare diseases: Knowledge-based success stories

Abstract

While more than 7000 rare diseases have been identified, only about 5 percent benefit from a licensed treatment. As the majority of these diseases is life threatening, these facts underscore the need for new drugs. Drug repositioning is an alternative strategy in drug development, which represents an attractive opportunity for rare diseases. Drug repositioning (also called drug repurposing, drug reprofiling or drug re-tasking) consists in identifying for an already approved or investigational drug a new use outside the scope of the original medical indication. Drug repositioning is considered in the field of orphan drugs as being a faster and somehow less costly strategy than traditional new drug development for pharmaceutical companies. While several successful repositioning cases have been discovered by serendipity, most successes straightly derive from the molecular characterization of the concerned disease. This short commentary is mainly dedicated to these rationally-based success stories.

Keywords: Alkaptonuria; Drug repositioning; Drug repurposing; Overgrowth syndrome; Progeria; Rare diseases.