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Home | Metabolism | Phenylketonuria (PKU) | Congress activities | The American College of Medical Genetics (ACMG) | ACMG 2026 poster: Sepiapterin responsiveness over 14 days in children and adults with phenylketonuria: Pooled results from three phase 3 clinical trials

ACMG 2026 poster: Sepiapterin responsiveness over 14 days in children and adults with phenylketonuria: Pooled results from three phase 3 clinical trials

This poster, originally given as an oral presentation at ACMG 2026, summarizes results from three Phase 3 clinical trials that assessed sepiapterin responsiveness in patients with phenylketonuria (PKU)

  • Learn more about the overall response rates to sepiapterin over a 14-day period in adults and children with PKU
  • Review the pooled efficacy data from three Phase 3 clinical trials: APHENITY, APHENITY Extension study and AMPLIPHY
  • Gain an overview of the safety profile of sepiapterin from these three studies
▼ 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 and was presented as an oral presentation at ACMG 2026.

The information in the materials was correct at the time of the events.

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