Repurposing as a strategy for orphan drug development, evidence from European approvals

Abstract

Introduction: Increased awareness of the value of drug repurposing has led to a number of initiatives to make discarded drug candidates available for screening. Repurposing has been suggested to be of considerable use in identifying drugs for the treatment of rare diseases. The 78 orphan drugs approved in Europe provide a useful set to analyze for evidence of repurposing.

Areas covered: Orphan drugs approved in Europe were classified as having been repurposed, as representing drugs specifically developed for orphan indications, or into those drugs that had been classified as orphan drugs after their utility had been established. Drugs were also classified as novel medical entities, biological products and natural products. The majority (60%) of approvals were of novel medical entities, including 17 of the 19 approved biological products. Natural products provided 10 of the approved drugs while 30 (38%) of the approvals were drugs repurposed from other indications.

Expert opinion: Identifying drugs for the treatment of rare diseases requires cost-effective discovery approaches. As the prevalence or incidence of the disease decreases, the need for lower cost approaches becomes more important. The systematic use of repurposing provides better opportunities for meeting such goals, and can facilitate subsequent development of the resulting drug(s).

Keywords::

• development strategy
• Europe
• orphan drugs
• repurposing

Notes

This box summarizes key points contained in the article.