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olipudase alfa-rpcp (Xenpozyme)
Indications:
- treatment of non-CNS manifestations of acid sphingomyelinasedeficiency in adults & children [1]
Contraindications:
- pregnancy
Dosage:
- adults: starting dose: 0.1 mg/kg IV infusion
- children: starting dose: 0.03 mg/kg IV infusion
*20 mg injection as lyophilized powder
Monitor:
- pregnancy test (reproductive capable females)
- baseline serum ALT, serum AST
Adverse effects:
- common (> 10%)
- headache, cough, diarrhea, hypotension, & ocular hyperemia
- elevated serum transaminases
- fetal malformations
Mechanism of action:
- lysosomal sphingomyelinase
General
sphingomyelinase, sphinogomyelin phosphodiesterase
metabolic agent (metabolic modifier)
References
- HIGHLIGHTS OF PRESCRIBING INFORMATION
Xenpozyme (olipudase alfa-rpcp) for injection, for intravenous use
https://products.sanofi.us/xenpozyme/xenpozyme.pdf
- Keam SJ
Olipudase Alfa: First Approval
Drugs. 2022 Jun;82(8):941-947.
PMID: 35639287
- Wikipedia: Olipudase alfa
https://en.wikipedia.org/wiki/Olipudase_alfa