No QT interval prolongation effect of sepiapterin: a concentration-QTc analysis of pooled data from phase 1 and phase 3 studies in healthy volunteers and patients with phenylketonuria.

ABSTRACT: Sepiapterin is an exogenously synthesized new chemical entity that is structurally equivalent to endogenous sepiapterin, a biological precursor of tetrahydrobiopterin (BH₄), which is a cofactor for phenylalanine hydroxylase. Sepiapterin is being developed for the treatment of hyperphenylalaninemia in pediatric and adult patients with phenylketonuria (PKU). This study employed concentration-QT interval analysis to assess QT prolongation risk following sepiapterin treatment. Data from three phase 1 studies and one phase 3 study were pooled for this analysis. Pediatric and adult PKU patients ≥ 2 years received multiple doses at 60 mg/kg and adult healthy volunteers received a single or multiple doses at 20 or 60 mg/kg. Time-matched triplicate ECG measurements and plasma samples for

SUBMITTER: Gao L 

PROVIDER: S-EPMC11739178 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature