Orphan drugs are designated drug substances that are intended to treat rare or orphan diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target.

Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from...