Table 1.
Prescribing summary of olipudase alfa (Xenpozyme®) in the treatment of non-CNS manifestations of acid sphingomyelinase deficiency in the EU [14] and USA [15]. Consult local prescribing information for further details
| What is the approved indication of olipudase alfa? | |
| EU: treatment of non-CNS manifestations of ASMD in adult and paediatric patients with type A/B or type B | |
| USA: treatment of non-CNS manifestations of ASMD in adult and paediatric patients | |
| How is olipudase alfa available and how should it be stored? | |
| Available as: lyophilised powder in a 20 mg single-dose vial for reconstitution and dilution for intravenous infusion | |
| Storage: refrigerate (do not freeze) original product at 2–8 °C; refrigerate reconstituted vials or diluted solutions (if not used immediately) at 2–8 °C for up to 24 h or store at room temperature (20–25 °C) for up to 12 h | |
| What is the recommended administration regimen and method for olipudase alfa? | |
| Dose-escalation phase | Adults: 0.1, 0.3, 0.3, 0.6, 0.6, 1 and 2 mg/kg at weeks 0, 2, 4, 6, 8, 10, and 12, respectively |
| Paediatric patients: 0.03, 0.1, 0.3, 0.3, 0.6, 0.6, 1 and 2 mg/kg at weeks 0, 2, 4, 6, 8, 10, 12 and 14, respectively | |
| Maintenance dose | 3 mg/kg every 2 weeks from week 14 in adults and week 16 in paediatric patients |
| Administration method | Increase infusion ratea incrementally in four steps and only in the absence of infusion-associated reactions, with the first three steps lasting for 20 min |
|
Adjust infusion volumesa patients with acid sphingomyelinase based on patient age and/or body weight | |
| How should olipudase alfa be used in special populations? | |
| Patients with abnormal kidney function | No dosage adjustment required |
| Patients with abnormal liver function | No dosage adjustment required |
| Elderly patients | No dosage adjustment required (based on limited data) |
| Breastfeeding patients | It is not known whether olipudase alfa is excreted in human milk, but a risk to newborns cannot be excluded based on animal studies |
| Discontinue breastfeeding or treatment based on benefits of breastfeeding for infant versus mother’s clinical need for treatment | |
| What other special warnings/precautions pertain to the use of olipudase alfa? | |
| Hypersensitivity reactions including anaphylaxis, infusion-associated reactions | Consider premedications (antihistamines, antipyretics, corticosteroids) |
| Severe reaction: discontinue treatment and initiate appropriate medical treatment | |
| Mild or moderate reaction: adjust infusion rate, temporarily withhold infusion, or adjust dosage | |
| Elevated transaminases | Monitor ALT and AST levels before and during treatment; adjust dosage based on transaminase levels or withhold treatment until levels return to baseline value |
| Risks during pregnancy | Do not initiate or escalate dosage at any time during pregnancy; base maintenance dosing decision on patient’s benefit-risk profile |
| Advise women of reproductive potential to use effective contraception during and for 14 days after the last dose of treatment | |
ALT alanine aminotransferase, ASMD acid sphingomyelinase deficiency, AST aspartate aminotransferase, CNS central nervous system
aConsult local prescribing information for the recommended infusion rates and volumes