Home | Metabolism | Phenylketonuria (PKU) | Congress activities | Society for Inherited Metabolic Disorders (SIMD) | SIMD 2026 poster: Sepiapterin responsiveness and long-term safety in participants with phenylketonuria aged less than or equal to 2 years: Interim results from the phase 3 APHENITY extension study

SIMD 2026 poster: Sepiapterin responsiveness and long-term safety in participants with phenylketonuria aged less than or equal to 2 years: Interim results from the phase 3 APHENITY extension study

This poster, presented at SIMD 2026, highlights interim results from the Phase 3 APHENITY Extension Study evaluating the long-term safety and responsiveness of sepiapterin treatment in children aged ≤2 years with phenylketonuria (PKU)

Learn more about the long-term safety and tolerability of the treatment sepiapterin in young children with PKU
Understand how baseline blood phenylalanine (Phe) levels may help predict treatment response, and the potential of early sepiapterin treatment to support metabolic control in young children with PKU
Review extension study findings demonstrating continued responsiveness to sepiapterin among participants receiving treatment

▼ 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 SIMD 2026.

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MED-ALL-PKU-2600120 | June 2026

SIMD 2026 poster: Sepiapterin responsiveness and long-term safety in participants with phenylketonuria aged less than or equal to 2 years: Interim results from the phase 3 APHENITY extension study

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