PTC Therapeutics Announces FDA Acceptance for Filing of NDA for Sepiapterin for the Treatment of Pediatric and Adult Phenylketonuria Patients
WARREN, N.J., Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). 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."
"NPKUA is deeply committed to improving the lives of individuals with PKU," said Catherine Warren, Executive Director, National PKU Alliance. "Our families have been anxiously waiting for new treatments that provide an opportunity to lessen their dependance on costly and burdensome diets. We are excited by today's news, as it brings us one step closer to providing another option for people of all ages with PKU to manage their condition."
The sepiapterin NDA is based on the highly statistically significant and clinically meaningful results from the phase 3 APHENITY trial. The results demonstrate a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in the subgroup of subjects with classical PKU. The majority of subjects (84%) achieved Phe control in accordance with treatment guidelines of
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