olipudase alfa-rpcp (Xenpozyme)

Indications

• treatment of non-CNS manifestations of acid sphingomyelinasedeficiency in adults & children

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

More general terms

• sphingomyelinase, sphinogomyelin phosphodiesterase
• metabolic agent (metabolic modifier)