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NCT05579548RECRUITING

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

Study Summary

This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding.

Conditions
Phenylketonuria, Maternal
Interventions / Drugs
Pegvaliase
View on ClinicalTrials.gov ↗
Sponsor
BMRN
BioMarin Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment50 participants
Primary Completion—
Lead SponsorBioMarin Pharmaceutical
Data Retrieved2026-04-11 02:55:23.531321+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 50 participants
Sponsor Catalyst Profile

BioMarin Pharmaceutical (BMRN) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

BMRN Full Pipeline →All Companies →
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