Phenylketonuria (PKU) Treatments
Find Phenylketonuria (PKU) Treatments
Brand Name
Sephience
Generic Name
Sepiapterin
View Brand Information FDA approval date: July 28, 2025
Form: Powder
What is Sephience (Sepiapterin)?
SEPHIENCE is indicated for the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria . SEPHIENCE is to be used in conjunction with a phenylalanine -restricted diet. SEPHIENCE is a phenylalanine hydroxylase activator indicated for the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria . SEPHIENCE is to be used in conjunction with a phenylalanine - restricted diet.
Approved To Treat
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Related Clinical Trials
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
Summary: The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.