PTC Therapeutics, Inc. (PTCT) Friday said that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for Sephience for the treatment of phenylketonuria, an inherited genetic disorder.
The New Drug Application (NDA) for sepiapterin is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of July 29, 2025. The drug is also under review in several other countries including Japan and Brazil.
"We look forward to initiating the European launch immediately following the adoption of this positive opinion by the European Commission," said Matthew B. Klein, Chief Executive Officer of PTC Therapeutics.
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