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Home | Metabolism | Phenylketonuria (PKU) | Publications | Author video: Highlights from the phase 3 APHENITY trial: Efficacy and safety of oral sepiapterin for children and adults living with PKU

Author video: Highlights from the phase 3 APHENITY trial: Efficacy and safety of oral sepiapterin for children and adults living with PKU

This is an author video developed to convey the key messages of the APHENITY trial manuscripts through the voices of two key authors

  • Learn more about the APHENITY study, which assessed the effect of sepiapterin on dietary Phe tolerance in a broad population of patients with phenylketonuria (PKU)
  • Understand the effectiveness of sepiapterin in patients with PKU in this double-blind, randomized, placebo-controlled trial
  • Gain an overview of the safety profile of sepiapterin in this study
▼ Sepiapterin is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-PTC923-2500012 | September 2025

Click here to access the APHENITY trial manuscript.

This author video was developed and funded by PTC Therapeutics.

Muntau A, Longo N, Ezgu F, et al. Lancet 2024; 404:1333–45

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