BioMarin’s Palynziq (pegvaliase-pqpz) Receives FDA Approval for Phenylketonuria (PKU) in Adults

BioMarin Reports Results of Brineura (cerliponase alfa) for Patients with CLN2 Disease Aged 3 to 16 yrs.


  • The approval is based on P-III PRISM-2 study results assessing Palynziq (pegvaliase-pqpz, 20mg qd or 40mg qd) in patients in ratio (2:1) with high level of phenylalanine (Phe)
  • The P-III PRISM-2 study resulted in maintaining blood Phe level vs PBO in patients
  • Palynziq (pegvaliase-pqpz, SC) is an enzyme substitution therapy of phenylalanine hydroxylase (PAH) enzyme helping in breaking down of Phe

Click here to read full press release/ article | Ref: Biomarin | Image: Biomarin