IntraBio Inc today announced that the US Food and Drug Administration has granted Orphan Drug Designation to Ursodeoxycholic Acid (UDCA) for the treatment of Niemann-Pick disease type C.
Niemann-Pick Disease Type C affects 1:100,000 live births, and is most commonly caused by dysfunction of the NPC1 protein leading to the accumulation of lipids in lysosomes, resulting in impaired cell function and cell death in various organs, leading to a spectrum of symptoms in NPC patients. The disease typically begins in early childhood and is chronic and progressive in nature; liver pathology, arising from the considerable lipid storage burden, is a common feature in NPC patients.
IntraBio, with its collaborators, has evaluated the effect of UDCA in pre-clinical and observational clinical studies to evaluate the therapeutic value of UDCA in Niemann-Pick disease Type C (NPC). The company intends to continue to investigate the compound of the treatment of rare, genetic diseases like NPC that have extremely high unmet medical needs.