GlaxoSmithKline Creates New Unit for Rare Diseases R&D

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ePT--the Electronic Newsletter of Pharmaceutical Technology

GSK launched a stand-alone unit that will specialize in the R&D and commercialization of medicines for rare diseases.

GlaxoSmithKline (GSK) launched a stand-alone unit that will specialize in the research and development (R&D) and commercialization of medicines for rare diseases. The operation, which will be led by Marc Dunoyer, president of GSK's Asia Pacific operations and chairman of its Japan group, will seek to "leverage existing capabilities and partnerships, and further establish in-licensing opportunities," according to a company press release.

"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 release. "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."

In 2009, GSK entered into collaborations with Prosensa (Leiden, The Netherlands) and JCR Pharmaceuticals (Hyogo, Japan), that 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, said in the press release. "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 being treated.