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BioWorld - Monday, January 19, 2026
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Home » PTC’s Sephience wins FDA nod for phenylketonuria
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PTC’s Sephience wins FDA nod for phenylketonuria

July 29, 2025
By Jennifer Boggs
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PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
BioWorld Regulatory Endocrine/metabolic Small molecule U.S. FDA

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