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PTC Therapeutics, Inc., a global biopharmaceutical company, announced that Sephience (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities.
"The European approval of Sephience is a great step in our efforts to bring this safe and highly effective therapy to children and adults affected by PKU worldwide," said Matthew B. Klein, M.D., chief executive officer of PTC Therapeutics. "The broad label supports that potential for Sephience to address all key PKU patient segments and become the new standard of care."
The European approval is based on the highly statistically significant results from the phase 3 APHENITY trial as well as evidence of durable treatment effect and the ability of study participants to liberalize their diet in the APHENITY long-term extension study.
The marketing authorization is applicable to all 27 European Union member states as well as Iceland, Norway and Liechtenstein. The Sephience European launch will be initiated in Germany in the first half of July.
A New Drug Application (NDA) for sepiapterin remains on schedule for its FDA target action date of July 29, 2025. Review of approval applications is ongoing in several other countries including Japan and Brazil.
Sephience (sepiapterin) is indicated for the treatment of hyperphenylalaninaemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Sephience acts as a dual pharmacological chaperone (sepiapterin and BH4 each with its own binding affinity to variant PAH), including PAH variants commonly found in PKU and known to be insensitive to BH4, to improve the activity of the defective PAH enzyme, achieving a high concentration of BH4 intracellularly. By enhancing the conformational stability of misfolded PAH enzyme and increasing the intracellular concentrations of BH4, Sephience is able to effectively reduce blood Phe levels. Sephience (sepiapterin) is approved in the European Economic Area. Sepiapterin is an investigational new drug in the United States.
Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe). If left untreated or poorly managed, Phe — an essential amino acid found in all proteins and most foods — can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders.
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