ELFABRIO Dosage & Rx Info | Uses, Side Effects

Elfabrio Generic Name & Formulations

Legal Class

General Description

Pegunigalsidase alfa-iwxj 2mg/mL; soln for IV infusion after dilution; preservative-free.

Pharmacological Class

Hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.

How Supplied

Single-dose vials (10mL)—1, 5, 10

Storage

Store refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze. Do not shake.

Manufacturer

Chiesi USA, Inc.

Generic Availability

Mechanism of Action

Elfabrio provides an exogenous source of alpha-galactosidase A. Elfabrio is internalized and transported into lysosomes where it is thought to exert enzymatic activity and reduce accumulated globotriaosylceramide (Gb3).

Elfabrio Indications

Indications

Treatment of confirmed Fabry disease.

Elfabrio Dosage and Administration

Adult

See full labeling. If enzyme replacement therapy (ERT)-experienced: consider similar premedications (if used) prior to first several Elfabrio infusions. May consider stepwise decrease or discontinuation of premedications after 4–6 infusions. If ERT-naive: consider pre-treating with antihistamines, antipyretics, and/or corticosteroids. Dose and infusion rates (initial 4–6 infusions) are based on actual body wt. Give 1mg/kg as IV infusion every 2 weeks. Infusion rates (ERT-experienced): <70kg: 0.83mL/min; 70–100kg: 1.39mL/min; >100kg: 2.78mL/min; (ERT-naive): <70kg: 0.63mL/min; 70–100kg: 1mL/min; >100kg: 1.38mL/min.

Children

Not established.

Elfabrio Contraindications

Not Applicable

Elfabrio Boxed Warnings

Boxed Warning

Hypersensitivity reactions. Anaphylaxis.

Elfabrio Warnings/Precautions

Warnings/Precautions

Have medical support and resuscitation equipment readily available. Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids prior to initiation. Discontinue immediately if severe hypersensitivity or infusion-associated reaction (IAR) occurs; may rechallenge with slower infusion rates. Interrupt temporarily or slow rate by 25–50% if mild to moderate hypersensitivity or IAR occurs. Monitor for presence of IgG and IgE anti-drug antibodies (ADA) if hypersensitivity reactions or IARs occur. Compromised cardiac function; monitor closely. Monitor SCr and urinary protein to creatinine ratio. Discontinue if glomerulonephritis is suspected. Patients with pre-existing ADA from prior ERT: monitor when switching from other ERT or agalsidase beta to Elfabrio. Pregnancy. Nursing mothers.

Elfabrio Pharmacokinetics

Metabolism

Catabolism.

Elimination

Half-life: 78.9±10.3 hours.

Elfabrio Interactions

Not Applicable

Elfabrio Adverse Reactions

Adverse Reactions

IARs, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, sinusitis; hypersensitivity, anaphylaxis, membranoproliferative glomerulonephritis.

Elfabrio Clinical Trials

Clinical Trials

The approval was based on data from 2 clinical trials: an open-label dose ranging study (ClinicalTrials.gov Identifier: NCT01678898) in patients who were enzyme replacement therapy (ERT)-naïve or who had not received ERT for more than 26 weeks and had a negative test for anti-pegunigalsidase alfa-iwxj IgG antibodies; as well as a phase 3, double-blind, and active-controlled trial (ClinicalTrials.gov Identifier: (NCT03566017) in ERT-experienced adults diagnosed with Fabry disease.

In the phase 3 trial, patients were randomly assigned to receive at least 1 dose of pegunigalsidase alfa (n=52) or agalsidase beta (n=25). The primary efficacy endpoint was the annualized rate of change in estimated glomerular filtration rate (eGFR slope) assessed over 104 weeks.

Findings showed that pegunigalsidase alfa was noninferior to agalsidase beta in controlling eGFR decline. The estimated mean eGFR slope was -2.4 and -2.3mL/min/1.73 m²/year on pegunigalsidase alfa and agalsidase beta respectively. The estimated treatment difference was -0.1mL/min/1.73 m²/year (95% CI, -2.3, 2.1).

Elfabrio Note

Notes

Report pregnancy exposure by calling (888) 661-9260.

Elfabrio Patient Counseling

Cost Savings Program

Chiesi Total Care & Patient Support

Elfabrio

PAGE CONTENTS