Elfabrio (pegunigalsidase alfa-iwxj) / Protalix, Chiesi - LARVOL DELTA

Feasibility and safety of pegunigalsidase alfa infusion during hemodialysis: first case report in a Fabry patient with kidney failure. (PubMed, J Nephrol) - No abstract available

Journal • Renal Disease

Pegunigalsidase alfa improves exercise tolerance and skeletal muscle bioenergetics in a Fabry disease mouse model (SSIEM 2026) - "PA treatment enhances exercise endurance and reduces fatigability in FD mice. This effect is likely mediated by improved mitochondrial energetics, rather than rescue of the muscle fiber metabolic switch. Indeed, the glycolytic component and higher power-output of FD skeletal muscle remain unchanged."

Preclinical • Fabry Disease • Genetic Disorders

Long-term safety and efficacy of pegunigalsidase alfa in patients who switched from agalsidase alfa (SSIEM 2026) - No abstract available

Clinical

Tolerability of 1 Hour Infusions of Pegunigalsidase Alfa in Fabry Disease: Data from the F60/BRILLIANCE Trial (SSIEM 2026) - No abstract available

Fabry Disease • Genetic Disorders

Real-world multicenter experience with four-week pegunigalsidase-alfa dosing in Fabry disease (SSIEM 2026) - "Clinical stability and good tolerability were observed, suggesting a potential reduction in treatment burden and improved adherence. Further studies with larger cohorts and longer follow-up are required to confirm long-term outcomes."

Clinical • Real-world • Real-world evidence • Atrial Fibrillation • Cardiovascular • Fabry Disease • Genetic Disorders • Otorhinolaryngology • Renal Disease

Long-term safety and efficacy of pegunigalsidase alfa in treatment-naive Fabry disease patients in F60/BRILLIANCE trial (SSIEM 2026) - P3 | "PA treatment for ≤10 years in ERT-naive FD patients was well-tolerated and associated with stable renal function and sustained lyso-Gb3 level reduction. Although patients with BL eGFR>110mL/min/1.73m² had steeper declines than those with BL eGFR110 is warranted."

Clinical • CNS Disorders • Fabry Disease • Genetic Disorders • Migraine

Flexible care in real life: experience with dosage regimens of pegunigalsidase alfa (SSIEM 2026) - "Sponsored by Chiesi"

Clinical

Long term clinical evidence with pegunigalsidase alfa (SSIEM 2026) - "Sponsored by Chiesi"

Clinical

Flexible by design: redefining treatment possibilities with Pegunigalsidase alfa (SSIEM 2026) - "Sponsored by Chiesi"

Evaluating the relationship between antidrug antibodies and efficacy and safety outcomes in patients with Fabry disease receiving enzyme replacement therapy: a systematic literature review. (PubMed, Orphanet J Rare Dis) - "This SLR found evidence that ADA positivity may be associated with increased IRR risk and may have a negative effect on some measures of ERT efficacy. Inconsistencies in the identified data were likely driven by study design differences, including prior ERT exposure, follow-up time, and ADA assessment protocols. Additional prospective studies with standardized ADA assessments and accounting for patient baseline characteristics and disease severity, are needed to better understand the clinical implications of ADA formation on ERT outcomes, including an assessment of causality."

Journal • Review • Cardiovascular • Fabry Disease • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

PEGASO: Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting (clinicaltrials.gov) - P=N/A | N=75 | Recruiting | Sponsor: Chiesi Italia | Not yet recruiting ➔ Recruiting

Enrollment open • Real-world evidence • Fabry Disease • Genetic Disorders

Pegunigalsidase Alfa in Fabry Disease Patients with Severe Infusion Reactions to Other Enzyme Replacement Therapy: A Spanish Multi-Center Experience (ERA 2026) - "This study aimed to evaluate the safety, tolerability, and clinical stability of PA in FD patients who previously discontinued agalsidase alfa or beta due to severe tolerability issues. Pegunigalsidase alfa represents a safe and effective "rescue" strategy for Fabry patients with history of severe IRRs. The PEGylation of the enzyme appears to successfully mask immunogenic epitopes, allowing for the continuation of essential therapy without compromising safety or biochemical control. These real-world data support PA as a viable alternative to overcome the limitations of other ERT in patients suffering severe infusion reactions"

Clinical • Fabry Disease • Genetic Disorders

FEASIBILITY AND SAFETY OF PEGUNIGALSIDASE ALFA INFUSION DURING HEMODIALYSIS IN A FABRY PATIENT WITH ESRD (ERA 2026) - No abstract available

Clinical • Renal Disease

Bright51: Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (clinicaltrials.gov) - P3 | N=29 | Completed | Sponsor: Chiesi Farmaceutici S.p.A. | Active, not recruiting ➔ Completed

Trial completion • Fabry Disease • Genetic Disorders

Kwangdong secures approval for Fabry disease treatment Elfabrio (Korea Biomedical Review)

Korea approval • Fabry Disease

GoPEG: German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa (clinicaltrials.gov) - P=N/A | N=60 | Active, not recruiting | Sponsor: Universität Münster | Recruiting ➔ Active, not recruiting

Enrollment closed • Fabry Disease • Genetic Disorders

Sex-based antibody subclass maturation drives direct enzymatic inhibition in fabry disease patients receiving enzyme replacement therapy. (PubMed, Front Mol Biosci) - "Subclass-specific cross-reactivity was assessed for agalsidase alfa, agalsidase beta, and pegunigalsidase alfa. Quantitative subclass-specific ADA and complement profiling reveals sex-specific IgG4 patterns, neutralizing capacity, and ERT-specific immunogenic differences, supporting its utility for personalized therapy in Fabry disease. A novel multiplex LC-MS/MS assay quantifies ADA subclasses and complement in Fabry disease, uncovering distinct IgG4 patterns and ERT-specific profiles that enhance understanding of treatment immunogenicity."

Journal • Fabry Disease • Genetic Disorders

Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding (clinicaltrials.gov) - P=N/A | N=10 | Recruiting | Sponsor: Chiesi Farmaceutici S.p.A. | Not yet recruiting ➔ Recruiting

Enrollment open • Fabry Disease • Genetic Disorders

Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce European Commission Approval of Additional Dosing Regimen of Every Four Weeks for Elfabrio (pegunigalsidase alfa) (Protalix Press Release) - "The EC approval is informed by results from an open-label, switch-over study, BRIGHT (formally PB-102-F50), designed to assess the adverse-event profile, efficacy, and pharmacokinetics (PK) of the alternative dosing regimen of pegunigalsidase alfa 2-mg/kg E4W for 52 weeks, and its ongoing open-label extension study CLI-06657AA1-03 (formerly PB-102-F51)."

EMA approval • Fabry Disease

PEGASO: Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting (clinicaltrials.gov) - P=N/A | N=75 | Not yet recruiting | Sponsor: Chiesi Italia | Initiation date: Jan 2026 ➔ Apr 2026

Real-world evidence • Trial initiation date • Fabry Disease • Genetic Disorders

Long-Term Safety and Efficacy of Pegunigalsidase Alfa in Patients with Fabry Disease: Results from the Phase 3 BRILLIANCE Extension Study (ACMG 2026) - P3 | "At baseline, most participants had received enzyme replacement therapy (71.1% agalsidase beta; 18.6% agalsidase alfa); 25.8% had anti-drug antibodies (ADAs) against PA, and median (range) baseline estimated glomerular filtration rate (eGFR) was 77.7 (24.4, 131.0) mL/min/1.73m². These findings demonstrate that long-term treatment with pegunigalsidase alfa 1 mg/kg E2W offers a sustained safety and efficacy profile in adults with FD over a median of nearly six years, supporting its role as a viable long-term therapy."

Clinical • P3 data • Fabry Disease • Genetic Disorders

A rapid method to reduce drug interferences for antibody measurements in pegunigalsidase alfa-treated patients with Fabry disease. (PubMed, Front Immunol) - "Alkaline pretreatment with NaOH was sufficient to eliminate up to 1 µg/ml agalsidase alfa or pegunigalsidase alfa in control sera. A second patient with pre-existing ADAs before pegunigalsidase alfa-initiation showed a massive induction of anti-PEG antibodies with inhibitory function. We present a rapid alkaline-treatment based method to overcome drug interferences to measure at least free antibodies in patients treated with pegunigalsidase alfa."

Journal • Fabry Disease • Genetic Disorders

Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for an Additional Dosing Regimen of Every Four Weeks for Elfabrio (pegunigalsidase alfa) in the EU (The Manila Times) - "The Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion following re-examination, which will be reviewed by the European Commission (EC), with a decision anticipated by March 2026....The CHMP opinion is based on results from an open-label, switch-over study, BRIGHT (formally PB-102-F50), designed to assess the safety, efficacy, and pharmacokinetics (PK) of the new dosing regimen of pegunigalsidase alfa 2 mg/kg E4W for 52 weeks, and its ongoing open-label extension study CLI-06657AA1-03 (formerly PB-102-F51, with a median exposure of almost 6 years). Further support is provided by an updated Population Pharmacokinetics (PopPK) model and exposure-response analysis, which leverage data from multiple clinical studies."

CHMP • Fabry Disease

Bright51: Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (clinicaltrials.gov) - P3 | N=29 | Active, not recruiting | Sponsor: Chiesi Farmaceutici S.p.A. | Trial completion date: Mar 2026 ➔ Jun 2026 | Trial primary completion date: Dec 2025 ➔ Apr 2026

Trial completion date • Trial primary completion date • Fabry Disease • Genetic Disorders

Lysosomal storage diseases in North America: a comprehensive review of enzyme therapies and unmet needs. (PubMed, Ther Adv Rare Dis) - "Newer formulations such as pegunigalsidase (Elfabrio®) use for FD (Fabry disease), exhibit lower affinity for developing ADA compared to other ERTs, offer reduced immunogenicity and safety profiles enhancement...This review draws from MEDLINE, Cochrane Reviews, and PubMed (1984-2025) using search terms such as LSDs, ERTs, rare diseases, Gaucher disease, Fabry disease, and others. Ongoing research and health policy reforms are essential to improve access, equity, and therapeutic outcomes for patients with LSDs."

Journal • Review • Fabry Disease • Gaucher Disease • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases