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Long-term comparative effectiveness of pegvaliase versus medical nutrition therapy with and without sapropterin in adults with phenylketonuria

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Burton et al. compared the long-term effectiveness of pegvaliase versus sapropterin and medical nutrition therapy (MNT), or MNT alone.

Pegvaliase more effectively reduced blood phenylalanine (Phe) concentrations in adults with PKU and blood Phe concentrations to >600 μmol/L compared with MNT, with or without sapropterin. The effectiveness of pegvaliase was found to increase over time and enable higher intact protein intake. This may contribute to better overall health and improve long-term outcomes for individuals with PKU.

The results of this study demonstrated that pegvaliase induced a sustained reduction in target blood Phe concentrations compared with MNT, with or without sapropterin.

Burton et al. 2024
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References:

Burton BK, Clague GE, Harding CO, Kucuksayrac E, Levy DG, Lindstrom K, Longo N, Malliot F, Muntau AC, Rutsch F, Zori RT.
Mol. Genet. Metab. 2024;141:108114.
DOI: 10.1016/j.ymgme.2023.108114