Treatment of phenylketonuria with sepiapterin

PL3801534T3Active Publication Date: 2026-07-06PTC THERAPEUTICS MP INC
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Patent Information

Authority / Receiving Office
PL · PL
Patent Type
Patents
Current Assignee / Owner
PTC THERAPEUTICS MP INC
Filing Date
2019-05-30
Publication Date
2026-07-06

AI Technical Summary

Technical Problem

Existing treatments for phenylketonuria (PKU) using tetrahydrobiopterin (BH4) are limited by its inability to cross the blood-brain barrier effectively, leading to insufficient intracellular BH4 levels in liver, kidney, and brain cells.

Method used

Administering sepiapterin with food delays absorption, allowing it to be converted into BH4 intracellularly, thereby increasing plasma, cerebrospinal fluid (CSF), and brain exposure of BH4.

Benefits of technology

Enhances BH4 plasma exposure and CSF levels, effectively treating phenylketonuria by increasing the maximum concentration and extent of BH4 exposure.

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