Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial

Publication: Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial

This research article summarized the results from the Phase 3 AMPLIPHY trial, which is the first study to compare the use of sepiapterin vs sapropterin in patients with phenylketonuria (PKU)

Learn about the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial
Understand the efficacy of sepiapterin vs sapropterin in patients with PKU
Gain an overview into the safety profile of sepiapterin vs sapropterin 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

Giżewska M, Inwood A, Tyčová R et al. Metabolism. 2026;178:156513.

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MED-ALL-PKU-2600079 | April 2026

Publication: Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial

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