Inborn errors of metabolism:

Indications for: XENPOZYME

Non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD).

Adult Dosage:

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 x 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 Dosage:

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 x 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. 

Boxed Warning:

Severe hypersensitivity reactions.

XENPOZYME 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 benefits/risks of retreating after a severe reaction. 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 >2xULN 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 Classification:

Hydrolytic lysosomal sphingomyelin-specific enzyme.

Adverse Reactions:

Headache, cough, diarrhea, hypotension, ocular hyperemia; children also: pyrexia, rhinitis, abdominal pain, vomiting, urticaria, nausea, rash, arthralgia, pruritus, fatigue, pharyngitis.

Generic Drug Availability:

NO

How Supplied:

Single-dose vial—1

Pricing for XENPOZYME

20mg vial (Qty: 1)
Appx. price $7051
GoodRx