Pegvaliase: First Global Approval

AbstractBioMarin Pharmaceutical is developing pegvaliase (PALYNZIQ ™) as a treatment for phenylketonuria, a genetic disorder caused by deficiency of phenylalanine hydroxylase which leads to neurotoxic accumulation of phenylalanine. Data from the phase III PRISM clinical trial program indicate treatment with pegvaliase is associated with sustained reductions in bl ood phenylalanine levels and sustained improvements in neurological sequelae in patients with phenylketonuria. Based on these positive results pegvaliase was recently approved in the US for adults with phenylketonuria who have uncontrolled blood phenylalanine concentrations on current treatment. Thi s article summarizes the milestones in the development of pegvaliase leading to this first approval.

http://link.springer.com/10.1007/s40259-018-0292-3

Source: BioDrugs - July 18, 2018 Category: Drugs & Pharmacology Source Type: research