PTC Therapeutics Says FDA to Review Proposed PKU Treatment

By Colin Kellaher

PTC Therapeutics on Tuesday said the Food and Drug Administration has accepted its application seeking approval of its proposed sepiapterin treatment for phenylketonuria, a rare, inherited metabolic disease better known as PKU.

The Warren, N.J., biopharmaceutical company said it expects the agency to provide a target action date for the application in about two weeks.

PTC earlier this year said the application covers pediatric and adult patients with PKU, including the full spectrum of ages and disease subtypes, and is based on highly statistically significant and clinically meaningful results from a Phase 3 study.

In a research note, analysts at William Blair said they expect sepiapterin will have a strong commercial launch in mid-2025, noting that new PKU patients are readily identified due to widespread newborn screening and that PTC will be able to leverage its established global commercial footprint for swift access to the drug.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

October 01, 2024 12:21 ET (16:21 GMT)

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