New unit for rare diseases R&D launched

February 12, 2010
Stephanie Sutton

Stephanie Sutton was an assistant editor at Pharmaceutical Technology Europe.

Pharmaceutical Technology Europe

GlaxoSmithKline (GSK) has launched a standalone unit that will specialize in the R&D and commercialization of medicines for rare diseases.

GlaxoSmithKline (GSK) has launched a standalone unit that will specialize in the R&D and commercialization of medicines for rare diseases. The operation, which will be led by Marc Dunoyer, GSK's President of Asia Pacific and Chairman of Japan, will seek to "leverage existing capabilities and partnerships, and further establish in-licensing opportunities," according to a company press statement.

"In addition to our existing discovery effort, alternative opportunities need to be explored to make treatments available for rare diseases," Dunoyer said in the press statement. "This complimentary 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."

In 2009, GSK entered into collaborations with Prosensa (The Netherlands) and JCR Pharmaceuticals (Japan), which both focus on therapeutics that could be used to treat orphan diseases.

"The entry into this new therapeutic area forms part of our strategy to deliver more products of value and improve returns in R&D through a focus on areas with a higher probability of success," Patrick Vallance, GSK's Senior Vice President of Drug Discovery, explained in the press statement. "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 specialized physicians to diagnose patients”.

According to GSK, more than 5500 rare diseases have been identified, but less than 10% are currently being treated.

www.gsk.com