GSK launches specialist rare diseases unit

GlaxoSmithKline (GSK) has launched a standalone unit to specialise in development and commercialisation of medicines for rare diseases.

The unit will be led by Marc Dunoyer, GSK's president of Asia Pacific and chairman of Japan. Dunoyer will work closely with GSK's senior vice president of Drug Discovery, Patrick Vallance.

This specialist unit will leverage existing capabilities and partnerships, such as those made by GSK with specialist companies, Prosensa and JCR Pharmaceuticals during 2009, and will establish further in-licensing opportunities.

As part of the agreement with JCR Pharmaceuticals, a Japanese developer and manufacturer of bioactive products, GSK obtained global rights to a number of enzyme replacement therapies that could, upon approval, be used to treat orphan diseases such as Hunter syndrome, Fabry disease and Gaucher disease. 

Marc Dunoyer said: "In addition to our existing discovery effort, alternative opportunities need to be explored to make treatments available for rare diseases. This complementary approach will combine our existing global expertise with specialist partners. Over time, this new unit has the potential to deliver multiple therapies responding to high medical needs of underserved populations of patients." 

Data from Orpha.net, a portal for rare diseases and orphan drugs, shows that over 5,500 rare diseases have been identified. Of these, less than 10 per cent are currently being treated. Many rare diseases are genetic in origin, start in childhood and cause lifelong debility and premature death.