Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• Sephience (sepiapterin) is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)-restricted diet.
• In participants of all ages with PKU and blood Phe concentration ≥360 μmol/L, the sepiapterin group showed a -63% (standard deviation [SD] 20) reduction in blood Phe concentration after 6 weeks, compared to a 1% (SD 29) change in the placebo group. The least squares mean change in blood Phe concentration was -395.9 μmol/L (SE 33.8) in favor of sepiapterin versus placebo, with a p-value <0.0001. No effectiveness outcomes were reported for specific subgroups within the study population.
• Among participants with PKU, treatment-emergent adverse events were reported in 59% (33/56) of those receiving sepiapterin and 33% (18/54) of those receiving placebo; mild gastrointestinal events occurred in 20% (11/56) of the sepiapterin group and 19% (10/54) of the placebo group. No deaths, serious, or severe adverse events were reported in either the sepiapterin or placebo groups.
• There is no population types or subgroups information available in the reviewed studies.