PTC announces FDA review date for NDA of sepiapterin

PTC Therapeutics announced that the FDA has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application, or NDA, for sepiapterin, consistent with a standard review timeline. The NDA was submitted with an indication for the treatment of pediatric and adult patients with phenylketonuria, or PKU, including all age groups and the full spectrum of disease subtypes. In addition, PTC announced that the results of the Phase 3 APHENITY trial were recently published in The Lancet, one of the most prestigious peer-reviewed medical journals. The sepiapterin NDA includes the results from the Phase 3 APHENITY trial discussed in The Lancet publication, as well as data from the ongoing APHENITY open-label extension study, which provides evidence of the durability of effect of sepiapterin and the ability of patients to liberalize their diet and still maintain control of phenylalanine levels. Sepiapterin, an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme. Phenylketonuria 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. If left untreated or poorly managed, phenylalanine – an essential amino acid found in all proteins and most foods – can build up to harmful levels in the body.