Drug repurposing is an innovative approach to drug development.

Instead of developing an entirely new compound to target a particular biological mechanism or protein, drug repurposing takes advantage of what is already known about the targets and mechanism of action of drugs that are already in use.

A single molecule can have multiple uses e.g. Everolimus, a drug originally indicated for use in preventing rejection in organ transplantation, is now used for multiple benign and cancerous tumours, the rare disease tuberous sclerosis complex (TSC), and seizure prophylaxis.

This multi-disease approach for one compound is being explored for Niemann Pick Disease – read more about this below.

The COVID-19 pandemic has accelerated interest in drug repurposing in recent years. As a result, multiple initiatives on national and global levels, including REMEDi4ALL, have been established or advanced to help continue this engagement and interest and to support those who are pursuing drug repurposing projects. Some of these initiatives can be found below:

• NHS medicines repurposing programme (UK)
• SIMPATHIC
• REPO4EU
• LifeArc
• NCATS New Therapeutic Uses (US)
• C-Path CURE ID (US)
• MHRA Innovative Licensing and Access Pathway (ILAP)
• EMA Safe and Timely Access to Medicine for Patients (STAMP) framework
• EveryCure

What makes drug repurposing innovative?

1. New tools (in silico, AI) making better use of existing data can efficiently generate more sound DR hypotheses that can be tested (in a streamlined platform).

2. An existing product can find wider application in other diseases, increasing impact and exploitation of the current drug.

3. The same active pharmaceutical ingredient (API)/compound can end up in multiple (bespoke) drug products (different formulations or dose strengths, tailored to the target population or individual – in the extreme coined as personalised medicine).

4. New intellectual property (IP) can be generated.

5. Combinations of existing drugs can be more effective (synergistic) where dose of individual drugs can be lowered and side effects reduced.

6.  Timely because certain early development steps (e.g. safety-toxicity profiling) could be avoided and production/market distribution is already in place.

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