PTC Therapeutics Files for FDA OK of Proposed PKU Treatment
By Colin Kellaher
PTC Therapeutics has filed for Food and Drug Administration approval of its proposed sepiapterin treatment of phenylketonuria, a rare, inherited metabolic disease better known as PKU.
PTC on Tuesday said the application covers pediatric and adult patients with PKU, including the full spectrum of ages and disease subtypes.
The Warren, N.J., biopharmaceutical company said its filing is based on the highly statistically significant and clinically meaningful results from a Phase 3 study.
PTC's European marketing application for sepiapterin for PKU is currently under review. There are an estimated 58,000 people with phenylketonuria globally, PTC said.
Write to Colin Kellaher at colin.kellaher@wsj.com
(END) Dow Jones Newswires
July 30, 2024 08:37 ET (12:37 GMT)
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