01:50 PM EDT, 07/30/2024 (MT Newswires) -- PTC Therapeutics ( PTCT ) said Tuesday it has submitted a new drug application for sepiapterin to the US Food and Drug Administration for the treatment of pediatric and adult patients with phenylketonuria, a rare metabolic disorder.

The application is supported by phase 3 trial results that showed a mean reduction of 63% in the levels of phenylalanine, an amino acid, in the overall treated population. There was also a reduction of 69% in phenylalanine levels in the subgroup of participants with classical phenylketonuria, the drugmaker said.

PTC Therapeutics ( PTCT ) said that its application for European marketing authorization is under review while marketing authorization applications for Japan and Brazil are anticipated to be filed later this year.

Price: 32.53, Change: +0.01, Percent Change: +0.03