Elfabrio

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

• Hypersensitivity reactions, including anaphylaxis: 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.
• Infusion-associated reactions: If severe IARs occur, discontinue Elfabrio and initiate appropriate medical treatment. 
• Membranoproliferative glomerulonephritis: Monitor serum creatinine and urinary protein to creatinine ratio. Discontinue Elfabrio if glomerulonephritis is suspected, until a diagnostic evaluation can be conducted.

The most common adverse reactions of Elfabrio include: 

• infusion-associated reactions,
• nasopharyngitis,
• headache,
• diarrhea,
• fatigue,
• nausea,
• back pain,
• pain in extremity, and
• sinusitis.

• 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.

Medical Support

• Appropriate medical support measures including cardiopulmonary resuscitation equipment should be readily available during Elfabrio administration.

Recommended Dosage and Administration

• 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.
• 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.

• 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.
• There are no data on the presence of pegunigalsidase alfa-iwxj in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Elfabrio and any potential adverse effects on the breastfed infant from pegunigalsidase alfa-iwxj or from the underlying maternal condition.