Sephience

<p>Sephience is a medicine used to treat hyperphenylalaninaemia (HPA, excessive blood levels of phenylalanine) in adults and children with phenylketonuria (PKU). PKU is an inherited condition where the amino acid phenylalanine (a building block for proteins) builds up in the blood to abnormally high levels, causing problems in the nervous system.</p><p>Hyperphenylalaninaemia is rare, and Sephience was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 May 2021. Further information on the orphan designation can be found on the EMA <a href="https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2435">website</a>.</p><p>Sephience contains the active substance sepiapterin. </p>