Posted by Michael Wonder on 07 May 2019
European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria in patients aged 16 years or older
6 May 2019 - First enzyme substitution therapy approved in Europe to treat the underlying cause of phenylketonuria in patients aged 16 years or older at doses up to 60 mg.
BioMarin Pharmaceutical today announced that the European Commission (EC) has granted marketing authorisation for Palynziq (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria 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.
The Palynziq EC approval was based on the totality of data from the Palynziq clinical development program including a Phase 3 pivotal study, PRISM-2, 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, compared to their baseline in the randomised discontinuation trial of 596.8 µmol/L and 410.9 µmol/L.
