Description
Pegvaliase pharmacokinetics differ among individuals, causing variable times to reach target blood Phe levels. This study aimed to model pegvaliase’s impact on blood Phe levels in PKU and identify markers to guide diet and dose adjustments after immune tolerisation.
Results showed that down-dosing was most likely to succeed when the preceding blood Phe was <30 µmol/L, higher pre-down-dosing Phe levels were linked to a higher failure rate and gradual, stepwise reduction was recommended until desired blood Phe levels were established. A staged approach to pegvaliase treatment includes achieving immune tolerance, liberalising diet, and gradually reducing dose.
References:
Harding CO, Whitehall KB, Lilienstein J, Sazova O, Lindstrom K, Levy DG, Burton BK. Genet Med. 2025;27(8):101459.