Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY extension study

Publication: Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY extension study

This research article reports the interim results from the phase 3 APHENITY extension study, which evaluates long-term treatment with sepiapterin in phenylketonuria (PKU) patients

Learn more about the long-term treatment of PKU with sepiapterin
Understand the effectiveness of long-term treatment with sepiapterin in these interim results from the ongoing, open-label, phase 3 APHENITY extension study
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

Van Spronsen F, Peters H, Margvelashvili L et al. Genet Med. 2026;12;28(4):101683

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MED-ALL-PKU-2600034 | April 2026

Publication: Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY extension study

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