Home | Metabolism | Phenylketonuria (PKU) | Publications | Publication: PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: A Phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study

Publication: PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: A Phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study

Published in Metabolism Clinical and Experimental, this research article presents the real-world efficacy of PTC923, compared with sapropterin, in a Phase 2 study of patients with phenylketonuria (PKU)

Understand the effect of PTC923 on blood phenylalanine (Phe) levels in patients with PKU
Find out about the relationship between different doses of PTC923 and blood Phe levels
Review the safety and efficacy of PTC923 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

Bratkovic D, Margvelashvili L, Tchan MC, et al. Metabolism 2022;128:155116

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MED-ALL-PKU-2200001 | October 2025

Publication: PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: A Phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study

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