Pegunigalsidase Alfa

Fabry disease is caused by deficiency of the lysosomal enzyme alpha-galactosidase A. ELFABRIO provides an exogenous source of alpha-galactosidase A.

• ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. ELFABRIO is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adults with confirmed Fabry disease.

• None. None.contraindicated

For pretreatment recommendations, see Full Prescribing Information. ( 2.1 ) Recommended dosage is 1 mg/kg every 2 weeks administered as an intravenous infusion. ( 2.2 ) For dosage and administration modifications due to hypersensitivity reactions or infusion-associated reactions (IARs), see Full Prescribing Information. ( 2.3 ) For instructions on preparation (including dilution), storage, and administration (including rates for the initial 4-6 infusions for ERT-experienced and ERT-naïve patients), see Full Prescribing Information. ( 2.4 , 2.5 , 2.6 ) 2. 1 Recommendations Prior to ELFABRIO Treatment Pretreatment In enzyme replacement therapy (ERT)-experienced patients, if pretreatment with antihistamines, antipyretics, and/or corticosteroids was used prior to ERT administration, consider similar pretreatment with these medications before the first several ELFABRIO infusions. After 4 to 6 ELFABRIO infusions, a stepwise decrease in the pretreatment medication dose(s) and/or discontinuation of the pretreatment medication(s) may be considered if treatment with ELFABRIO was tolerated. In ERT-naïve patients, prior to ELFABRIO administration, pre-treating with antihistamines, antipyretics, and/or corticosteroids may be considered [see Warnings and Precautions ( 5.1 , 5.2 )] . Medical Support Appropriate medical support measures including cardiopulmonary resuscitation equipment should be readily available during ELFABRIO administration. 2.

Infusion-Associated Reactions: If severe IARs occur, discontinue ELFABRIO and initiate appropriate medical treatment. ( 5.2 ) Membranoproliferative Glomerulonephritis: Monitor serum creatinine and urinary protein to creatinine ratio. Discontinue ELFABRIO if glomerulonephritis is suspected, until a diagnostic evaluation can be conducted. ( 5.3 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Hypersensitivity reactions including anaphylaxis have been reported in ELFABRIO-treated patients. In clinical trials, 20 (14%) of ELFABRIO-treated patients experienced hypersensitivity reactions. In these trials, 4 ELFABRIO-treated patients (3%; 1 naïve to enzyme replacement therapy (ERT) and 3 ERT-experienced patients) experienced anaphylaxis during the initial infusion and were positive for anti-pegunigalsidase alfa-iwxj IgE antibodies (referred to as IgE ADA) [see Adverse Reactions ( 6.1 6. 1 ) and Clinical Pharmacology ( 12.6 ) ] . The risk of pegunigalsidase alfa-iwxj-related hypersensitivity may be increased in certain patients with pre-existing ADA from prior ERT [ see Use In Specific Populations ( 8.6 )]. Anaphylaxis (reported as Type I hypersensitivity reaction, hypersensitivity reaction, or bronchospasm) occurred within 5 to 40 minutes of the start of the initial infusion. Signs and symptoms included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema.

The following clinically significant adverse reactions are described elsewhere in labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions ( 5.1 ) ] Infusion-Associated Reactions (IARs) [see Warnings and Precautions ( 5.2 )] Membranoproliferative Glomerulonephritis [see Warnings and Precautions ( 5.3 )] Most common adverse reactions (≥15%) are: infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Chiesi USA, Inc. at 1-888-661-9260 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trial of another drug and may not reflect the rates observed in clinical practice. Adverse Reactions From Trial 2 The safety of ELFABRIO in adults with confirmed Fabry disease who had been previously treated with agalsidase beta was evaluated in Trial 2 which included a total of 52 ELFABRIO-treated patients (29 male, 23 female aged 20 to 60 years old) with Fabry disease [see Clinical Studies ( 14 )] . Patients received 1 mg/kg of ELFABRIO given intravenously every 2 weeks for at least 104 weeks.

8.1 Pregnancy Risk Summary There are no available data on ELFABRIO use in pregnant females to evaluate a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes; however, as an enzyme replacement, ELFABRIO is not expected to cause adverse outcomes . Animal reproduction studies have been conducted with pegunigalsidase alfa-iwxj in pregnant rats and rabbits. No adverse effects on embryofetal development were observed in pregnant rats intravenously administered pegunigalsidase alfa-iwxj twice per week at exposures up to 3.6 times that of the maximum recommended human dose (MRHD) (based on area under the concentration-time curve (AUC)). Maternal toxicity was observed in pregnant rabbits intravenously administered pegunigalsidase alfa-iwxj twice per week at doses that were ≥ 3.2 times the MRHD (based on human equivalent dose) [ see Data ] . The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. There is a pregnancy safety study for ELFABRIO.

Metabolism

The pharmacokinetics (PK) of pegunigalsidase alfa-iwxj were evaluated in adult patients with Fabry disease and are presented as mean (standard deviation, SD) unless otherwise specified. The pharmacokinetics of pegunigalsidase alfa-iwxj in plasma following intravenous infusion of ELFABRIO 1 mg/kg every 2 weeks in adult treatment-naïve patients with Fabry disease are summarized in Table 4.

• May 9, 2023BLABLA761161Chiesi Farmaceutici Spa

1. 1Fatigue3412%
2. 2Off Label Use3211%
3. 3Nausea227.5%
4. 4Pain217.1%
5. 5Ill-defined Disorder155.1%
6. 6Product Dose Omission Issue155.1%
7. 7Pyrexia155.1%
8. 8Infusion Related Reaction144.7%
9. 9Asthenia124.1%
10. 10Vomiting124.1%
11. 11Chills113.7%
12. 12Chest Pain103.4%
13. 13Pain In Extremity103.4%
14. 14Product Administration Interrupted103.4%
15. 15Burning Sensation82.7%

The 10 most recently updated of 15 ClinicalTrials.gov registrations naming Pegunigalsidase Alfa as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
  Completed · Phase 3 · Interventional · 29 enrolled · Chiesi Farmaceutici S.p.A.
  NCT03614234updated 2026-05-22
• German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
  Active not recruiting · Observational · 60 enrolled · Universität Münster
  NCT06095713updated 2026-04-30
• Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
  Completed · Phase 3 · Interventional · 97 enrolled · Chiesi Farmaceutici S.p.A.
  NCT03566017updated 2026-03-24
• A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
  Recruiting · Phase 2 · Phase 3 · Interventional · 22 enrolled · Chiesi Farmaceutici S.p.A.
  NCT06328608updated 2026-03-19
• 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
  Recruiting · Observational · 10 enrolled · Chiesi Farmaceutici S.p.A.
  NCT06941025updated 2026-03-19
• Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
  Recruiting · Phase 2 · Phase 3 · Interventional · 16 enrolled · Chiesi Farmaceutici S.p.A.
  NCT05710692updated 2026-03-18
• Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
  Not yet recruiting · Observational · 75 enrolled · Chiesi Italia
  NCT07109375updated 2026-02-24
• A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
  Recruiting · Observational · 100 enrolled · Chiesi Farmaceutici S.p.A.
  NCT06663358updated 2025-11-14
• Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
  Approved for marketing · Expanded access · Chiesi Farmaceutici S.p.A.
  NCT04552691updated 2024-07-31
• Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
  Completed · Phase 3 · Interventional · 30 enrolled · Protalix
  NCT03180840updated 2023-09-13

Frequently asked questions

How does Pegunigalsidase Alfa work?

Fabry disease is caused by deficiency of the lysosomal enzyme alpha-galactosidase A. ELFABRIO provides an exogenous source of alpha-galactosidase A.

What is Pegunigalsidase Alfa used for?

According to FDA labeling, Pegunigalsidase Alfa carries indications including: ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. ELFABRIO is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adults with confirmed Fabry disease.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Pegunigalsidase Alfa?

Pegunigalsidase Alfa is classified as Enzymes, Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme, Glycosphingolipid Synthesis Increase.

What are the brand names for Pegunigalsidase Alfa?

Pegunigalsidase Alfa is marketed under brand names including Elfabrio.

What are the contraindications for Pegunigalsidase Alfa?

Pegunigalsidase Alfa labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.