sepiapterin

Sepiapterin is an orally administered small-molecule precursor in the tetrahydrobiopterin pathway being developed primarily for phenylketonuria. It was originally developed by Censa Pharmaceuticals and later acquired by PTC Therapeutics through the Censa merger. In the provided sources it is described as the lead asset for PKU and other orphan metabolic indications, with positive Phase 3 topline data reported by PTC. Pharmacologically, sepiapterin serves as a biochemical precursor of tetrahydrobiopterin, a critical cofactor for phenylalanine hydroxylase and other aromatic amino acid hydroxylases; by increasing intracellular tetrahydrobiopterin availability, it is intended to improve residual phenylalanine hydroxylase activity and lower phenylalanine levels in patients with PKU. Preclinical literature in the provided materials also describes sepiapterin as a precursor of BH4 that can modulate nitric oxide synthase coupling and endothelial function.

Brand names

Sephience

Other names

sepiapterin