Medically reviewed on December 11, 2017.
See full prescribing information for complete boxed warning. 1
Anaphylaxis has occurred with vestronidase alfa-vjbk administration, as early as the first dose; therefore, appropriate medical support should be readily available when vestronidase alfa-vjbk is administered. 1
Closely observe patients during and for 60 minutes after vestronidase alfa-vjbk infusion. 1
Immediately discontinue the vestronidase alfa-vjbk infusion if the patient experiences anaphylaxis.1
Vestronidase alfa-vjbk is an enzyme.
Uses for Vestronidase Alfa-vjbk
Vestronidase alfa-vjbk has the following uses:
Vestronidase alfa-vjbk is a recombinant human lysosomal beta glucuronidase indicated in pediatric and adult patients for the treatment of mucopolysaccharidosis VII (MPS VII, Sly syndrome). 1
Vestronidase alfa-vjbk has the following limitations of use:
The effect of vestronidase alfa-vjbk on the central nervous system manifestations of MPS VII has not been determined.1
Vestronidase Alfa-vjbk Dosage and Administration
Vestronidase alfa-vjbk is available in the following dosage form(s) and strength(s):
Injection: 10 mg/5 mL (2 mg/mL) in a single-dose vial).1
It is essential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:
The recommended dosage is 4 mg/kg administered every two weeks as an intravenous infusion.1
Premedication with a non-sedating antihistamine with or without an antipyretic is recommended 30 to 60 minutes prior to the start of the infusion.1
Administer the infusion over approximately 4 hours. In the first hour of infusion, infuse 2.5% of the total volume. After the first hour, the rate can be increased to infuse the remainder of the volume over 3 hours as tolerated.1
For additional information on preparation, administration (including rate of infusion by dose and body weight), and storage, see the full prescribing information.1
Cautions for Vestronidase Alfa-vjbk
Anaphylaxis to vestronidase alfa-vjbk was reported in 2 of 20 patients in the clinical program. These reactions occurred during vestronidase alfa-vjbk infusion and were observed as early as the first dose of vestronidase alfa-vjbk for one patient. Manifestations included respiratory distress, cyanosis, decreased oxygen saturation, and hypotension. The two patients with anaphylaxis to vestronidase alfa-vjbk during the clinical trials had one occurrence each and tolerated subsequent infusions of vestronidase alfa-vjbk, without recurrence.1
Anaphylaxis can be life-threatening. Vestronidase alfa-vjbk should be administered under the supervision of a healthcare professional with the capability to manage anaphylaxis. Patients should be observed for 60 minutes after vestronidase alfa-vjbk administration. If severe systemic reactions occur, including anaphylaxis, immediately discontinue the vestronidase alfa-vjbk infusion and provide appropriate medical treatment. Prior to discharge, inform patients of the signs and symptoms of anaphylaxis and instruct them to seek immediate medical care if symptoms occur. Consider the risks and benefits of re-administering vestronidase alfa-vjbk following anaphylaxis.1
Risk Summary: There are no available data on vestronidase alfa-vjbk use in pregnant women to determine a drug-associated risk of adverse developmental outcomes. In animal reproduction studies, vestronidase alfa-vjbk administered intravenously to pregnant rats and rabbits during the period of organogenesis showed no maternal toxicity or adverse developmental outcomes at doses causing maternal serum exposures (AUC) up to 1.6 and 10 times, respectively for rats and rabbits, the exposure at the recommended human dose. 1
The estimated background risk of major birth defects and miscarriage for 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.1
Animal Data: In animal reproduction studies, vestronidase alfa-vjbk administered intravenously to pregnant rats (once a week) and rabbits (once every 3 days) during the period of organogenesis showed no adverse developmental outcomes at doses up to 20 mg/kg. The 20 mg/kg dose in rats and rabbits provides approximately 1.6 and 10 times the human exposure (AUC) of 57.9 hr•mcg/mL at the 4 mg/kg dose administered once every other week, respectively. 1
There are no data on the presence of vestronidase alfa-vjbk 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 vestronidase alfa-vjbk and any potential adverse effects on the breastfed infant from vestronidase alfa-vjbk or from the underlying maternal condition.1
The safety and effectiveness of vestronidase alfa-vjbk have been established in pediatric patients less than 18 years of age.1
Clinical trials of vestronidase alfa-vjbk did not include any patients aged 65 and over. It is not known whether elderly patients respond differently from younger patients.1
Common Adverse Effects
Most common adverse reactions (≥1 patient) are: infusion site extravasation, diarrhea, rash, anaphylaxis, infusion site swelling, peripheral swelling, and pruritus.)1
It is essential that the manufacturer's labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:
Please see product labeling for drug interaction information.
Mechanism Of Action
Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage.1
Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.1
Advice to Patients
Advise patients and caregivers that anaphylaxis has occurred with vestronidase alfa-vjbk administration. Inform patients of the signs and symptoms of anaphylaxis, and have them seek immediate medical care should signs and symptoms occur.1
AHFS First Release. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.
Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.
Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.
2 mg/1 mL
Ultragenyx Pharmaceutical Inc.
AHFS Drug Information. © Copyright 2018, Selected Revisions December 11, 2017. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.
1. Ultragenyx Pharmaceutical Inc.. MEPSEVII (vestronidase alfa) INTRAVENOUS prescribing information. 2017 Nov.
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