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Xenpozyme Generic Name & Formulations
Legal Class
General Description
Pharmacological Class
How Supplied
Single-dose vial—1
Manufacturer
Generic Availability
NO
Mechanism of Action
ASMD is a lysosomal storage disease that results from reduced activity of the enzyme acid sphingomyelinase (ASM), caused by pathogenic variants in the sphingomyelin phosphodiesterase 1 gene. ASM degrades sphingomyelin to ceramide and phosphocholine. The deficiency of ASM causes an intra-lysosomal accumulation of sphingomyelin (as well as cholesterol and other cell membrane lipids) in various tissues. Xenpozyme provides an exogenous source of ASM.
Xenpozyme Indications
Indications
Non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD).
Xenpozyme Dosage and Administration
Adult
See full labeling. Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids. Administer via IV infusion every 2 weeks. If BMI (≤30): use actual body weight; (≥30): calculate adjusted body weight (kg) = (actual height in m)2 × 30. ≥18yrs: Dose escalation phase: (Week 0): 0.1mg/kg; (Weeks 2 and 4): 0.3mg/kg; (Weeks 6 and 8): 0.6mg/kg; (Week 10): 1mg/kg; (Week 12): 2mg/kg; (Week 14): 3mg/kg. Maintenance phase: (Week 16 and onward): 3mg/kg.
Children
See full labeling. Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids. Administer via IV infusion every 2 weeks. If BMI (≤30): use actual body weight; (≥30): calculate adjusted body weight (kg) = (actual height in m)2 × 30. 0-17yrs: Dose escalation phase: (Week 0): 0.03mg/kg; (Week 2): 0.1mg/kg; (Weeks 4 and 6): 0.3mg/kg; (Weeks 8 and 10): 0.6mg/kg; (Week 12): 1mg/kg; (Week 14): 2mg/kg; (Week 16): 3mg/kg. Maintenance phase: (Week 18 and onward): 3mg/kg.
Xenpozyme Contraindications
Not Applicable
Xenpozyme Boxed Warnings
Boxed Warning
Hypersensitivity reactions including anaphylaxis.
Xenpozyme Warnings/Precautions
Warnings/Precautions
Have medical resuscitation equipment readily available. Discontinue immediately and treat if a severe hypersensitivity reaction (eg, anaphylaxis) or severe infusion-associated reaction occurs. Consider temporarily holding or slowing the infusion rate, and/or reducing Xenpozyme dose if a mild to moderate hypersensitivity reaction or a mild to moderate infusion-associated reaction occurs. Consider testing for IgE ADA and other lab testing (eg, serum tryptase and complement activation) in those with severe hypersensitivity reactions. Obtain baseline ALT/AST levels within 1 month prior to initiation, within 72 hours prior to any infusion during dose escalation, or prior to the next scheduled infusion upon resuming after a missed dose. Withhold treatment temporarily or adjust dose if transaminase levels are elevated above baseline and >2×ULN prior to the next scheduled dose until liver transaminases return to baseline value. Pregnancy: not recommended (risk of fetal malformations during dosage initiation or escalation, or at any time during pregnancy); if needed, exclude status prior to initiation. Advise females of reproductive potential to use effective contraception during and for 14 days after the last dose if treatment is discontinued. Nursing mothers.
Xenpozyme Pharmacokinetics
Distribution
Mean (SD) volume of distribution: 13 (2) L (in adults with ASMD).
Elimination
Half-life: 32–38 hours. Mean (SD) clearance: 0.33 (0.07) L/h.
Xenpozyme Interactions
Not Applicable
Xenpozyme Adverse Reactions
Adverse Reactions
Headache, cough, diarrhea, hypotension, ocular hyperemia; children also: pyrexia, rhinitis, abdominal pain, vomiting, urticaria, nausea, rash, arthralgia, pruritus, fatigue, pharyngitis.
Xenpozyme Clinical Trials
Xenpozyme Note
Not Applicable
Xenpozyme Patient Counseling
See Literature
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