The European Commission has approved BioMarin’s Palynziq (pegvaliase injection) for the treatment of phenylketonuria (PKU) in patients aged 16 years or older.
The governing body granted marketing authorisation at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) - an amino acid that is found in most forms of protein - concentrations in patients who have inadequate blood Phe control despite prior management.
The 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.
The approval is based on the Phase III PRISM-2 study, which showed that a group of patients taking either 20 mg or 40 mg of Palynziq maintained mean blood Phe levels at 553.0 μmol/L and 566.3 μmol/L respectively after eight weeks.
"Palynziq is a new and promising treatment for the PKU community”, said Dr Amaya Bélanger-Quintana, head of the Metabolic Department of the University Hospital Ramon y Cajal, Madrid, Spain.
“Many adult PKU patients struggle daily with their special diet, leading many of them to relax or stop their treatment despite knowing the negative consequences this will have on to their well-being. Palynziq provides a new opportunity for adult PKU patients to attain the metabolic control their doctors and they want for themselves."
PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain and is marked by an inability to break down Phe. It affects approximately 50,000 diagnosed patients in the developed world, and in Europe, approximately 1 in every 10,000 newborn babies are affected by this disease.