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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The American College of Medical Genetics (ACMG) | ACMG 2025 symposium: Highlights from the phase 3 APHENITY trial: Efficacy and safety of oral sepiapterin for children and adults living with PKU

ACMG 2025 symposium: Highlights from the phase 3 APHENITY trial: Efficacy and safety of oral sepiapterin for children and adults living with PKU

This symposium, presented at ACMG 2025, summarizes the results from the APHENITY trial, which assessed the effect of sepiapterin on dietary Phe tolerance in a broad population of patients with phenylketonuria (PKU)

  • Learn more about the APHENITY study from two expert speakers
  • 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

This symposium was developed and funded by PTC Therapeutics for ACMG 2025.

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MED-ALL-PKU-2500001 | October 2025

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