Sepiapterin FDA Approval Status
Last updated by Judith Stewart, BPharm on Oct 30, 2024.
FDA Approved: No
Generic name: sepiapterin
Company: PTC Therapeutics, Inc.
Treatment for: Phenylketonuria
Sepiapterin is a precursor of tetrahydrobiopterin (BH4) in development to reduce blood phenylalanine levels in pediatric and adult patients living with phenylketonuria.
- Phenylketonuria (PKU) is a rare, metabolic disease caused by a defect in the gene that helps create the phenylalanine hydroxylase (PAH) enzyme needed to break down phenylalanine. Phenylalanine is an essential amino acid found in all proteins and most foods, and if it builds up to harmful levels in the body can cause severe and irreversible disabilities such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Diagnosis of phenylketonuria usually takes place during newborn screening programs.
- Sepiapterin works to reduce blood phenylalanine levels in patients with phenylketonuria by increasing activity of the PAH enzyme. It has a dual mechanism of action as a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (a critical cofactor of PAH) and also has an independent chaperone effect, protecting against PAH misfolding to enhance the enzyme function.
Development timeline for sepiapterin
Further information
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