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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The Society for the Study of Inborn Errors of Metabolism (SSIEM) | SSIEM 2026 poster: EPIPHENY study design: Phase 3b study of long-term neurocognitive outcomes in sepiapterin-treated children with PKU

SSIEM 2026 poster: EPIPHENY study design: Phase 3b study of long-term neurocognitive outcomes in sepiapterin-treated children with PKU

This poster, presented at SSIEM 2026, outlines the design of the EPIPHENY Phase 3b study, which evaluates the effects of long-term sepiapterin on neurocognitive function in children with phenylketonuria (PKU)

  • Understand the study design and key inclusion and exclusion criteria of the ongoing EPIPHENY study
  • Assess how primary, secondary, and exploratory endpoints will be used to evaluate the efficacy and safety of long-term sepiapterin treatment, including effects on neurocognitive function and health-related quality of life
  • Review the demographics and clinical characteristics of participants enrolled to date
▼ 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 poster was developed and funded by PTC Therapeutics for SSIEM 2026.​

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MED-ALL-PKU-2600178 | September 2026

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