BioMarin Expands PKU Dominion With EU Approval Of Palynziq

BioMarin Pharmaceutical Inc. has received approval for Palynziq (pegvaliase) to treat adults with the rare disease phenylketonuria (PKU) in the European Union. The injectable pegylated recombinant enzyme product is the first enzyme substitution therapy for the condition, and was approved in the US in May 2018. BioMarin markets the only two products approved for PKU in the US and EU.

PKU is a genetic metabolic disorder that causes toxic levels of phenylalanine, an amino acid contained in protein-rich foods, to accumulate in the brain and blood, potentially leading to seizures...