Drugs / pegunigalsidase alfa
last change Apr 2026 re-read 2 minutes ago

pegunigalsidase alfa

Protein / enzyme biologic

Developed for
Fabry disease
Investigated by
Chiesi Farmaceutici S.p.A. · Protalix · ICON plc · Chiesi USA, Inc.

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Elfabrio 2023-05-04 europa.eu

Trials 11 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/22 trials
Phase 1/2 NCT01981720 Jan 2014 → Aug 2020 Fabry disease Protalix Completed No outcome recorded
Phase 1/2 NCT01678898 Oct 2012 → Mar 2016 Fabry disease Protalix Completed No outcome recorded
Phase 2/32 trials
Phase 2/3 NCT06328608 Jul 2025 → Oct 2028 expected Fabry disease Chiesi Farmaceutici S.p.A. Recruiting No outcome recorded
Phase 2/3 NCT05710692 Aug 2023 → Oct 2027 expected Fabry disease Chiesi Farmaceutici S.p.A. Recruiting No outcome recorded
Phase 36 trials
Phase 3 NCT03614234 Nov 2018 → Apr 2026 Fabry disease Chiesi Farmaceutici S.p.A. Completed No outcome recorded
Phase 3 NCT03566017 Sep 2018 → Jan 2025 Fabry disease Chiesi Farmaceutici S.p.A. Completed No outcome recorded
Phase 3 NCT03180840 Jul 2017 → Aug 2020 Fabry disease Protalix Completed No outcome recorded
Phase 3 NCT02921620 Jul 2017 → Jul 2018 Fabry disease Protalix Withdrawn No outcome recorded
Phase 3 NCT03018730 May 2017 → Dec 2019 Fabry disease Protalix Completed No outcome recorded
Phase 3 NCT02795676 Jun 2016 → Oct 2021 Fabry disease Protalix Completed No outcome recorded
Phase not stated1 trial
NCT04552691 Fabry disease Chiesi Farmaceutici S.p.A. Approved for marketing No outcome recorded

Evidence & citations 8 cited values

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

FieldValueCited text
Known as pegunigalsidase alfa “A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE)” NCT05710692
6

“This study aims to learn how safe pegunigalsidase alfa (PRX-102 for short) is and how it works at treating Fabry disease in children and adolescents.” NCT06328608

NCT03566017

NCT03614234

NCT03018730

NCT03180840

NCT04552691

Known as Elfabrio ChEMBL registry synonym — accepted as the source's own label CHEMBL4297801
Known as Pegunigalsidasa alfa ChEMBL registry synonym — accepted as the source's own label CHEMBL4297801
Known as Pegunigalsidase alfa-iwxj ChEMBL registry synonym — accepted as the source's own label CHEMBL4297801
Known as PRX-102 “pegunigalsidase alfa (PRX-102) is a drug made using genetic engineering techniques and manufactured using cultured tobacco cells.” NCT05710692
6

“This is an open label switch over study to assess the safety and efficacy of PRX-102 (pegunigalsidase alfa).” NCT03018730

NCT06328608

NCT01981720

NCT02921620

NCT01678898

NCT02795676

Action Restore “developed as enzyme replacement therapy (ERT) for Fabry disease (FD)” PMID 30834538 Apr 2019
Modality Protein / enzyme biologic “PRX-102, an enzyme being developed as a long-term enzyme replacement therapy” NCT01678898
Route Intravenous “receive PRX-102 as an intravenous infusion every 2 weeks for 12 weeks (3 months)” NCT01678898