FDA Approves label expansion to allow maximum dose of 60 mg for Palynziq injection for treatment of adults with PKU.- BioMarin

BioMarin Pharmaceutical Inc. announced that the FDA has approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of 60 mg with Palynziq (pegvaliase-pqpz) Injection for treatment of adults with Phenylketonuria (PKU). Previously, the maximum dose was 40 mg. In the Phase III PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq. The label expansion includes the addition of longer-term efficacy data demonstrating sustained phenylalanine (Phe) lowering out to three years and over six years of data further supporting a well-characterized safety profile. The labeling was updated to allow individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 umol/L), taking into account patient tolerability to Palynziq and dietary protein intake. Palynziq is indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 umol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. Palynziq is BioMarin's second approved treatment for this serious condition. BioMarin also recently announced that the Company had dosed the first participant in the global Phearless Phase 1/II study of BMN 307, an investigational gene therapy for people with PKU, and potentially its third approved treatment. BMN 307 has Fast Track Designation in the US, as well as Orphan Drug Designation in the U.S. and E.U.