Drug repositioning. When development of novel drugs fails?
Drug repositioning or repurposing is a
strategy by which new or additional value is generated from a drug by targeting
diseases other than those for which it was originally intended. Repositioning
of launched or failed drugs has opened up a new source of revenue to large,
medium and small Pharma companies as well as attracting venture capital funding,
and is expected to generate up to $20 billion in annual sales in 2012. Drug repositioning
is not a new strategy and there are a number of examples of drugs that have
been successfully repositioned such as gemcitabine and sildenafil (Table 1).
These and many other examples have addressed an unmet medical need in selected
patient groups as well as creating substantial value for the company that
repositioned the drug. One of the most interesting and extreme examples of
repositioning is thalidomide, which was launched in 1957 as a sedative but was
later found to be responsible for severe birth defects in children when used by
pregnant women to alleviate morning sickness. After additional scientific
inquiry the drug has since been shown to be safe in selected patient groups and
to be very effective in pain relief in leprosy and Kaposi’s sarcoma, generating
$528M in revenue per year.
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