PTC Therapeutics announced on October 14, 2024, that the U.S. Food and Drug Administration (FDA) has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin. This timeline is consistent with a standard review process for the treatment of pediatric and adult patients with phenylketonuria (PKU).
In a significant validation of its clinical data, the results of the Phase 3 APHENITY trial for sepiapterin were recently published in The Lancet, a prestigious peer-reviewed medical journal. This publication underscores the promising and transformative nature of the data collected for sepiapterin.
The sepiapterin NDA includes these Phase 3 results, along with data from the ongoing APHENITY open-label extension study. This extension study provides evidence of the durability of sepiapterin's effect and its ability to allow patients to liberalize their diet while maintaining control of phenylalanine levels.
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