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

F. K. Trefz; Anita Inwood; Renata Tyčová; Suresh Vijay; Olivia Fjellbirkeland; Francjan van Spronsen; Eva Venegas‐Moreno; Laura Guilder; Alberto Burlina; Heidi Peters; Murray A. Potter; Urh Groselj; A. Chakrapani; K. Dokoupil; François Maillot; Frank Rutsch; Jean-Baptiste Arnoux; Michel Tchan; Kim M. Ingalls; Zhenming Zhao; Catalina Hughes; Neil Smith; Ania C. Muntau

doi:10.1016/j.metabol.2026.156513

Key Points

Sepiapterin significantly lowers blood Phe levels compared to sapropterin, highlighting its efficacy.
No new safety signals were reported during the trial, ensuring patient safety.
This active-controlled trial compares two therapies in phenylketonuria, a genetic disorder affecting amino acids.
Findings suggest sepiapterin may be a better treatment option, warranting further clinical exploration.

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Implication

Phase 3 trial demonstrates sepiapterin lowers blood Phe more than sapropterin, indicating potential benefits.

Abstract

Sepiapterin was superior to the highest approved dose of sapropterin in lowering blood Phe. No new safety signals were observed. The trial was registered in the UK Clinical Study Registry, ISRCTN, on January 29, 2024 (ID number, ISRCTN79102999; https://www.isrctn.com/ISRCTN79102999).

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Cite This Study

Trefz et al. (2026) studied this question.

synapsesocial.com/papers/69a75d06c6e9836116a266d4 https://doi.org/10.1016/j.metabol.2026.156513

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

Key Points

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Implication

Abstract

Discussion

Cite This Study

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

Key Points

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Implication

Abstract

Discussion

Cite This Study

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