Sapropterin for phenylketonuria: A Japanese post-marketing surveillance study.

ABSTRACT:

Background

The aim of this study was to assess the long-term safety and efficacy of sapropterin in a real-world setting in Japanese patients with tetrahydrobiopterin (BH4)-responsive phenylketonuria.

Methods

This post-marketing surveillance study enrolled all of the patients in Japan with confirmed BH4-responsive PKU who were administrated sapropterin between July 2008 and October 2017. Patients were observed at least every 3 months during follow up, with key data collected on treatment exposure/duration, effectiveness according to physician's judgement, serum phenylalanine levels, and adverse events.

Results

Of 87 enrolled patients, 85 patients (male, 42.4%; outpatients, 96.5%) were included in the safety and efficacy analysis sets. Treatment started at age <4 years

SUBMITTER: Tamura M 

PROVIDER: S-EPMC9305189 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature