FDA approval for first enzyme therapy to treat PKU

25 May 2018

The US Food and Drug Administration has granted approval 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.

Developed by BioMarin Pharmaceutical (Nasdaq: BMRN), 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 the second approved treatment for this important condition for BioMarin, whose shares moved up as much as 4.2% to $92.00 in after-hours trading on Thursday. Its currently marketed drug is Kuvan (sapropterin dihydrochloride), but that is coming up for generic competition.

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