PTC Therapeutics announced the FDA has accepted for filing the New Drug Application, NDA, of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria. A Prescription Drug User Fee Act, PDUFA, target action date is expected to be provided in the Day 74 Letter. “The FDA filing acceptance for sepiapterin is a critical milestone toward bringing this potential important therapy to children and adults living with PKU in the United States,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “The clinical trial data, including the evidence of enabling diet liberalization, support that sepiapterin can provide significant benefit to PKU patients, helping to meet the persistent, significant unmet need in this community.”
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