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Drug

Pegunigalsidase alfa

Approved · EMA
Late-stage development

Also known as Elfabrio.

RxNorm2637446UNII8M7V7Q6537
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease

Clinical trial2026-04-13Completed · ClinicalTrials.gov

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

Clinical trial2026-03-24Results posted · ClinicalTrials.gov

Phase 3 Open-Label Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2mg/kg IV Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy Fabrazyme® or Replagal™

Clinical trial2023-09-13Results posted · ClinicalTrials.gov

Approval: Elfabrio (EMA)

Regulatory2023-05-04EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Pegunigalsidase alfa
Aliases & brands
Elfabrio
RxNorm CUI
2637446
UNII
8M7V7Q6537
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2023-05-04
Latest approval
2023-05-04
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2023-05-04Approval
Approval: Elfabrio (EMA)

Indication: Elfabrio is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

3 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
3
registered trials across all phases
LATEST COMPLETION 2026
3
Late-stage (III+)
3
Completed
PHASE DISTRIBUTIONn = 3
Phase 33

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease

CompletedPH IIIChiesi Farmaceutici S.p.A.n=292026

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

CompletedPH IIIChiesi Farmaceutici S.p.A.n=972025

Phase 3 Open-Label Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2mg/kg IV Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy Fabrazyme® or Replagal™

CompletedPH IIIProtalixn=302020
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.