Pharming's Prodarsan gets US orphan status

Netherlands biotechnology firm company Pharming Group NV says that its wholly-owned subsidiary DNage BV has received a notice from the US Food  and Drug Administration that its product Prodarsan has been awarded an  Orphan Drug designation for the treatment of Cockayne Syndrome.  Prodarsan is a combination of two small molecules, formulated for oral  use, which are believed to reduce the accumulation of DNA-damage, the  underlying biochemical cause of Cockayne Syndrome.

Cockayne syndrome, the initial target of Prodarsan, is a rare genetic  disease in which children suffer from accelerated (or premature) aging,  while developing severe aging diseases. Progeria diseases are a group of  genetic premature aging conditions that manifest in several forms that  are genetically and clinically similar although not identical.