Sepiapterin (Sephience):New FDA-Approved Therapy for Phenylketonuria (PKU)
Introduction On July 28, 2025, the US Food and Drug Administration (FDA) approved a breakthrough therapy called Sephience, which contains the active compound sepiapterin. This approval marked a significant advance in the treatment of hyperphenylalaninemia (HPA), a condition known as phenylketonuria (PKU), in adults and pediatric patients. PKU is a rare genetic metabolic disorder that …