Palynziq approved to treat phenylketonuria by European Commission

BioMarin Pharmaceutical Inc announced that the European Commission (EC) has granted marketing authorization for Palynziq (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options.

Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first enzyme substitution therapy approved in Europe to target the underlying cause of PKU by helping the body to break down Phe. In addition, the EC acknowledged that the Phase III trial and extension study is suggestive of an improvement in inattention and mood symptoms.