BioMarin Receives the US FDA’s Approval Allowing Maximum Dose of 60mg with Palynziq (pegvaliase-pqpz) for PKU

Shots:

• The US FDA has approved the sBLA to increase the maximum allowable dose of 60mg with Palynziq for PKU. Previously, the maximum dose was 40mg
• The label expansion is based on OLE study out to 3yrs. demonstrating that 66% had a blood Phe level ≤360 μmol/L consistent with the Phe target ACMG recommended guidelines @2yrs. of treatment and 50% had blood Phe levels ≤120 μmol/L @2yrs. 75%, 66% & 48% had a blood Phe ≤600, 360, and 120 μmol /L, respectively @3yrs. of treatment
• Additional, safety data with over 6yrs. of follow up remains consistent with the previous safety profile of Palynziq irrespective of dose. Moreover, BioMarin has dosed the first participant in the global Phearless P-I/II study of BMN 307 for PKU
  
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