GSK looks to rare diseases research
pharmafile | February 5, 2010 | News story | Research and Development | GlaxoSmithKline, Orphan
GlaxoSmithKline has launched a new specialist unit to research and develop medicines for rare diseases.
The company has identified 5,500 rare diseases and estimate that less than 10% are currently being treated.
Many of the diseases are genetic in origin, start in childhood and cause lifelong debility and pre-mature death and, despite their rarity, in total may affect 6-8% of the population.
Marc Dunoyer, GSK’s president of Asia Pacific and chairman of Japan, will lead this new operation, working closely with Patrick Vallance, GSK’s senior vice president of drug discovery. The new unit will seek to leverage existing capabilities and partnerships and establish further in-licensing opportunities.
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. Overtime, this new unit has the potential to deliver multiple therapies responding to high medical needs of underserved populations of patients.”
Patrick Vallance said: “The entry into this new therapeutic area forms part of GSK’s strategy to deliver more products of value and improve returns in R&D through a focus on areas with a higher probability of success.
“The risk associated with product discovery and development in rare diseases is generally lower than other disease areas as disease definitions are very clear and clinical trials tend to be small with robust endpoints. In most cases the molecular target is known, making it easier for specialised physicians to diagnose patients”.
In 2009, GSK entered into strategic colloborations with Prosensa and JCR Pharma. The alliance with Prosensa is set to develop new therapies in correcting gene expression in diseases with large unmet medical needs.
The agreement with Japan-based JCR has seen GSK obtain global rights to a number of enzyme replacement therapies that have the potential treat several orphan diseases.
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