Olipudase alfa

No UK SPC (eMC) in the bundle — figures are from US prescribing information (XENPOZYME, Genzyme, label date 2025-12-11). Adult dose escalation regimen, all given every 2 weeks by IV infusion: Week 0 (first dose) 0.1 mg/kg; Week 2 0.3 mg/kg; Week 4 0.3 mg/kg; Week 6 0.6 mg/kg; Week 8 0.6 mg/kg; Week 10 1 mg/kg; Week 12 2 mg/kg; Week 14 3 mg/kg (the escalation phase includes the first 3 mg/kg dose, which is also the recommended maintenance dose). Weight basis: use actual body weight if BMI is 30 or less; if BMI is greater than 30 use adjusted body weight (kg) = (actual height in m) squared x 30. Before initiation: obtain baseline ALT and AST within 1 month prior to starting, and verify pregnancy status in females of reproductive potential. Consider premedication with antihistamines, antipyretics and/or corticosteroids. Appropriate medical support including cardiopulmonary resuscitation equipment should be readily available during administration. Assess ALT/AST within 72 hours prior to any infusion during dose escalation, and prior to the next scheduled infusion when resuming after a missed dose. A dose is considered missed if not given within 3 days of the scheduled date: in the escalation phase after 1 missed dose give the last tolerated dose then resume escalation; after 2 consecutive missed doses give 1 dose below the last tolerated dose then resume escalation; in the maintenance phase after 1 missed dose give the maintenance dose; after 2 consecutive missed doses give 1 dose below the maintenance dose then resume maintenance; for 3 or more consecutive missed doses the label directs re-initiation of the escalation regimen (full instruction truncated in the fetched text — clinician to check the current label). Therapy should be directed in consultation with physicians knowledgeable in the management of ASMD.

US labelling only (no UK SPC in bundle). Paediatric patients 0 to 17 years. Recommended starting dose is 0.03 mg/kg, with escalation every 2 weeks by IV infusion: Week 0 0.03 mg/kg; Week 2 0.1 mg/kg; Week 4 0.3 mg/kg; Week 6 0.3 mg/kg; Week 8 0.6 mg/kg; Week 10 0.6 mg/kg; Week 12 1 mg/kg; Week 14 2 mg/kg; Week 16 3 mg/kg (recommended maintenance dose). Same body-weight rule as adults (actual body weight if BMI 30 or less; adjusted body weight = (height in m) squared x 30 if BMI greater than 30). Compared with adults, a higher percentage of paediatric patients experienced treatment-related serious adverse reactions, anaphylaxis, hypersensitivity reactions and infusion-associated reactions within 24 hours of infusion. Verify every paediatric dose against a children's formulary and specialist metabolic advice before administration.