GSK may venture into rare disease treatments

LONDON GlaxoSmithKline has identified more than 5,500 rare diseases it could potentially target as part of a new drug-development initiative, the British drug maker announced Thursday.

GSK said it would start a new standalone business unit specializing in creating drugs for treating rare diseases, many of them genetic disorders that can cause lifelong disability or premature death. The company plans to work with Netherlands-based Prosensa and Japan-based JCR Pharmaceuticals, which it entered collaborations with in 2009.

The partnership with Prosensa, which started in October, focuses on drugs based on nucleic acid for Duchenne muscular dystrophy. With JCR, GSK will help develop drugs for Hunter syndrome, Fabry disease and Gaucher disease.

“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,” GSK SVP drug discovery Patrick Vallance stated. “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.”