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
  
   ­ Ref: BioMarin | Image: BioMarin Careers