Drugs / Pegvaliase
last change Aug 2026 re-read 2 minutes ago

Pegvaliase

Protein / enzyme biologic

Developed for
phenylketonuria
Investigated by
BioMarin Pharmaceutical

Regulatory milestones approvals, filings & regulatory actions · 4 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansions (2) US (FDA) PALYNZIQ 2020-10-06 – 2026-02-27 fda.gov
Approved EU (EMA) Palynziq 2019-05-03 europa.eu
Approved US (FDA) PALYNZIQ 2018-05-24 fda.gov

Trials 7 · a red edge is where a trial was stopped

201020112012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 24 trials
Phase 2 NCT01560286 May 2012 → Sep 2013 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 2 NCT01212744 Mar 2011 → Apr 2015 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 2 NCT00924703 Jan 2010 → Jan 2019 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 2 NCT00925054 Sep 2009 → Jun 2015 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 32 trials
Phase 3 NCT05270837 Jun 2022 → Jan 2025 overdue phenylketonuria BioMarin Pharmaceutical Active not recruiting No outcome recorded
Phase 3 NCT03694353 Sep 2018 → Jan 2021 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 41 trial
Phase 4 NCT06780332 Feb 2025 → Aug 2026 overdue phenylketonuria BioMarin Pharmaceutical Active not recruiting No outcome recorded
Also used as a comparator or background therapy in 4 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT00634660 Comparator May 2008 → Apr 2009 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 32 trials
Phase 3 NCT01889862 Comparator Jul 2013 → Jan 2016 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 3 NCT01819727 Comparator May 2013 → Nov 2015 phenylketonuria BioMarin Pharmaceutical Completed No outcome recorded
Phase 41 trial
Phase 4 NCT07477691 Background May 2026 → Sep 2027 expected phenylketonuria BioMarin Pharmaceutical Recruiting No outcome recorded

Evidence & citations 10 cited values

Every value below carries the sentence it was read from. 13 sources stand behind the page.

FieldValueCited text
Known as Pegvaliase Pegvaliase is approved to reduce phenylalanine (Phe) levels for people with phenylketonuria (PKU).” PMID 34826353 Nov 2021
3

NCT03694353

NCT07477691

NCT05270837

Known as BMN 165 ClinicalTrials.gov intervention name — accepted as the source's own label NCT01560286
1

NCT01819727

Known as Palynziq ClinicalTrials.gov intervention name — accepted as the source's own label NCT06780332
Known as Peg pal ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802
Known as Pegvaliasa ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802
Known as Pegvaliase pqpz ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802
Known as Phenylase ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802
Known as rAvPAL-PEG “BMN 165 (rAvPAL-PEG)” NCT01560286
4

NCT01212744

NCT00924703

NCT00925054

NCT00634660

Modality Protein / enzyme biologic “PEGylated recombinant Anabaena variabilis phenylalanine ammonia lyase” PMID 32593547 Jun 2020
1

CHEMBL4297802

Route Subcutaneous subcutaneous injection” NCT00634660