Posted by Michael Wonder on 25 May 2018
BioMarin receives standard approval for Palynziq (pegvaliase-pqpz) injection for treatment of adults with phenylketonuria, a rare enetic Disease
24 May 2018 - First enzyme therapy to treat phenylketonuria.
BioMarin today announced that BioMarin received standard approval from the U.S. FDA for Palynziq (pegvaliase-pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. Palynziq is BioMarin's second approved treatment for this important condition.
Palynziq significantly and substantially reduced blood Phe levels as demonstrated in the pivotal Phase 3 PRISM-2 study, which met the primary endpoint of change in blood Phe compared with placebo (p<0.0001).
