Description
Rohr et al. investigated the impact of pegvaliase on dietary intake using data from Phase 3 PRISM clinical trials.
Pegvaliase treatment resulted in most individuals with phenylketonuria (PKU) reaching and maintaining target blood phenylalanine (Phe) levels. 50% of patients achieved physiological normal blood Phe levels of ≤120 μmol/L by month 15 while consuming a normal diet. No evidence of protein malnutrition was noted.
The results of this study demonstrated that pegvaliase treatment reduced reliance on medical protein and allowed participants to increase both intact protein and total protein intake while lowering blood Phe.
References:
Rohr F, Burton B, Dee A, Harding CO, Lilienstein J, Lindstrom K, MacLeod E, Rose S, Singh R, vun Calcur S, Whitehall K
Mol. Genet. Metab. 2024;141:108122
DOI: 10.1016/j.ymgme.2023.108122