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General Information
Elfabrio (pegunigalsidase alfa-iwxj) is a PEGylated enzyme replacement therapy (ERT). It is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.
Elfabrio is specifically indicated for the treatment of adults with confirmed Fabry disease.
Elfabrio is supplied as an injection for intravenous administration.
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.
The recommended dosage of Elfabrio
, based on actual body weight, is 1 mg/kg
administered by intravenous infusion every 2 weeks.
The initial recommended Elfabrio
infusion rates for ERT-experienced or ERT-naïve
patients are based on actual body weight, per the drug prescribing label.
If one or more doses are missed, restart Elfabrio
treatment as soon as possible,
maintaining the 2 week interval between infusions thereafter. Do not double a dose to
compensate for a missed dose.
Mechanism of Action
Fabry disease is caused by deficiency of the lysosomal enzyme alpha-galactosidase A. Elfabrio is a PEGylated enzyme replacement therapy. 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).
Side Effects
Adverse effects associated with the use of Elfabrio may include, but are not limited to, the following:
- infusion-associated reactions
- nasopharyngitis
- headache
- diarrhea
- fatigue
- nausea
- back pain
- pain in extremity
- sinusitis
The Elfabrio drug label comes with the following Black Box Warning: Hypersensitivity reactions, including anaphylaxis, may occur with Elfabrio. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue Elfabrio immediately and initiate appropriate medical treatment.
Clinical Trial Results
The FDA approval of Elfabrio was based on a comprehensive clinical development program in more than 140 patients with up to 7.5 years of follow up treatment. It has been studied in both ERT-naïve and ERT-experienced patients, including a head-to-head trial that met its primary endpoint with Elfabrio demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline.