Publication: Phase I clinical evaluation of CNSA-001 (sepiapterin), a novel pharmacological treatment for phenylketonuria and tetrahydrobiopterin deficiencies, in healthy volunteers

This research article in Molecular Genetics and Metabolism Reportsreports results from a Phase 1 clinical study of CNSA-001 (sepiapterin), an agent being investigated for potential treatment of BH4 deficiencies and phenylketonuria (PKU) 

  • Results from a randomized, double-blind, dose-ranging, Phase 1 clinical trial of 83 healthy subjects who were administered CNSA-001 or placebo are presented and discussed
  • The pharmacokinetic profile, including plasma levels of sepiapterin and BH4 following administration of CNSA-001 is reported here, along with adverse events associated with treatment

Smith N, Longo N, Levert K, et al.Mol Genet Metab Rep. 2019;126(4):406412 

Sepiapterin is an investigational drug currently in development for PKU.

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MED-ALL-PKU-2100026 | January 2024
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