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

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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Publication Details

Journal
Metabolism
Published
2026-01-28
DOI
https://doi.org/10.1016/j.metabol.2026.156513
Primary Topic
Metabolism and Genetic Disorders
Type
article
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article

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

Olivia Fjellbirkeland, Eva Venegas‐Moreno, Kim M. Ingalls, Zhenming Zhao et al.
Metabolism
Metabolism and Genetic Disorders
article

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

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

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).

MetabolismVol. 178
Université de Tours (FR), Royal Children's Hospital (AU), University Medical Center Groningen (NL), University of Padua (IT), Inserm (FR), Universität Hamburg (DE), University of Groningen (NL), University of Toronto (CA), Université Paris Cité (FR), Charles University (CZ), Great Ormond Street Hospital (GB), Hospital for Sick Children (CA), Ljubljana University Medical Centre (SI), McMaster Children's Hospital (CA), Rigshospitalet (DK), Westmead Hospital (AU), Birmingham Children's Hospital (GB), University Hospital Münster (DE), University Medical Center Hamburg-Eppendorf (DE), Kinderkrebs-Zentrum Hamburg (DE), PTC Therapeutics (United States) (US), Hospital Universitario Virgen del Rocío (ES), Hospital Universitario Ramón y Cajal (ES), Institut des Maladies Génétiques Imagine (FR), Children's Health Queensland Hospital and Health Service (AU), University College London (GB), Hamilton Health Sciences (CA), Pomeranian Medical University (PL), University of Hong Kong (HK)
Good health and well-being
Openalex Percentile: Top 70%
Metabolism and Genetic Disorders
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