Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study

Longo, N.; Zori, R.; Wasserstein, M. P.; Vockley, J.; Burton, B. K.; Decker, C.; Li, M.; Lau, K.; Jiang, J.; Larimore, K.; Thomas, J. A.

Orphanet J Rare Dis. 2018 Jul 6; 13(1):108


BACKGROUND: Deficiency of phenylalanine hydroxylase causes phenylketonuria (PKU) with elevated phenylalanine (Phe) levels and associated neuropsychiatric and neurocognitive symptoms. Pegvaliase (PEGylated phenylalanine ammonia lyase) is an investigational agent to lower plasma Phe in adults with PKU. This study aimed to characterize the long-term efficacy, safety, and immunogenicity of pegvaliase in adults with PKU. METHODS: PAL-003 is an ongoing, open-label, long-term extension study of the pegvaliase dose-finding parent phase 2 studies. Participants continued the dose of pegvaliase from one of three parent studies, with dose adjustments to achieve a plasma Phe concentration between 60 and 600 mumol/L. RESULTS: Mean (standard deviation [SD]) plasma Phe at treatment-naive baseline for 80 participants in the parent studies was 1302.4 (351.5) mumol/L. In the 68 participants who entered the extension study, plasma Phe decreased 58.9 (39)% from baseline, to 541.6 (515.5) mumol/L at Week 48 of treatment. Plasma Phe concentrations

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