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PTC Therapeutics Announces Sepiapterin NDA Submission to FDA
WARREN, N.J., July 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin NDA to the U.S. FDA. The
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"WARREN, N.J., July 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin NDA to the U.S. FDA. The NDA submission is for the treatment of pediatric and adult patients with phenylketonuria (PKU), including the full spectrum of ages and disease subtypes.\n\n\"This NDA submission is an important step in bringing this therapy to children and adults living with PKU in the U.S.,\" said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. \"The robust sepiapterin clinical trial data, including Phe tolerance results, support the potential of sepiapterin to meet the persistent unmet medical need for the large number of children and adults with PKU.\"\nThe 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 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","length":1586,"tagName":"div"}]