Home | Metabolism | Phenylketonuria (PKU) | Congress activities | European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria (E.S.PKU) | E.S.PKU 2026 poster: Achievement of normal-range blood phenylalanine (less than 120 μmol/l) with sepiapterin in PKU: APHENITY extension interim analysis

E.S.PKU 2026 poster: Achievement of normal-range blood phenylalanine (less than 120 μmol/l) with sepiapterin in PKU: APHENITY extension interim analysis

This poster, presented at E.S.PKU 2026, reports on the interim results from the APHENITY Extension Study, which evaluated long-term blood phenylalanine (Phe) control with sepiapterin treatment in people with phenylketonuria (PKU)

Assess the proportion of study participants achieving normal-range blood Phe levels (<120 µmol/L) during long-term treatment with sepiapterin, including across age groups and BH₄ responsiveness status
Evaluate the impact of sepiapterin on dietary protein tolerance and dietary flexibility while maintaining metabolic control during diet liberalization
Understand the long-term safety and tolerability of sepiapterin and its potential to support sustained blood Phe control in people with PKU

▼ 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 E.S.PKU 2026.

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MED-ALL-PKU-2600186 | September 2026

E.S.PKU 2026 poster: Achievement of normal-range blood phenylalanine (less than 120 μmol/l) with sepiapterin in PKU: APHENITY extension interim analysis

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