IntravenousAcid sphingomyelinase deficiencyAdult: For the treatment of non-CNS manifestation cases in patients with type A/B or type B disease: For patients with BMI ≤30 kg/m 2, use the actual body weight; for patients with BMI >30 kg/m 2, use the optimal body weight (refer to detailed product guideline for the weight calculation).
Dose escalation phase:
| Dose number (timing) |
Dosage |
| 1st dose (Day 1/Week 0) |
0.1 mg/kg |
| 2nd dose (Week 2) |
0.3 mg/kg |
| 3rd dose (Week 4) |
0.3 mg/kg |
| 4th dose (Week 6) |
0.6 mg/kg |
| 5th dose (Week 8) |
0.6 mg/kg |
| 6th dose (Week 10) |
1 mg/kg |
| 7th dose (Week 12) |
2 mg/kg |
| 8th dose (Week 14) |
3 mg/kg |
Maintenance phase: 3 mg/kg every 2 weeks. All doses are administered via IV infusion. Dose reduction or interruption may be required according to individual safety or tolerability (refer to detailed product guidelines). Dosing recommendations for missed doses vary according to the number of missed infusions and the specific dosing phase; infusion rate and duration vary according to dosage. Refer to specific or detailed product guidelines for further missed dose management and infusion administration instructions. Child: For patients with BMI ≤30 kg/m 2, use the actual body weight; for patients with BMI >30 kg/m 2, use the optimal body weight (refer to detailed product guideline for the weight calculation).
Dose escalation phase:
| Dose number (timing) |
Dosage |
| 1st dose (Day 1/Week 0) |
0.03 mg/kg |
| 2nd dose (Week 2) |
0.1 mg/kg |
| 3rd dose (Week 4) |
0.3 mg/kg |
| 4th dose (Week 6) |
0.3 mg/kg |
| 5th dose (Week 8) |
0.6 mg/kg |
| 6th dose (Week 10) |
0.6 mg/kg |
| 7th dose (Week 12) |
1 mg/kg |
| 8th dose (Week 14) |
2 mg/kg |
| 9th dose (Week 16) |
3 mg/kg |
Maintenance phase: 3 mg/kg every 2 weeks. All doses are administered via IV infusion. Dose reduction or interruption may be required according to individual safety or tolerability (refer to detailed product guidelines). Dosing recommendations for missed doses vary according to the number of missed infusions and the specific dosing phase; infusion rate and duration vary according to dosage. Refer to specific or detailed product guidelines for further missed dose management and infusion administration instructions.
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Powder for solution for IV infusion: Allow vials to reach room temperature for 20-30 minutes.
Adults and children:
| Vial strength |
Reconstitution |
| 4 mg |
Reconstitute each vial with 1.1 mL of sterile water for inj. |
| 20 mg |
Reconstitute each vial with 5.1 mL of sterile water for inj to make a concentration of 4 mg/mL. |
Direct the diluent flow onto the inside wall of the vial and gently roll or tilt vials to avoid foaming. Withdraw the appropriate dose and required volume from the vial(s), then dilute with NaCl 0.9% inj, as follows:
Dilution for adults:
| Dosage |
Final volume (mL) |
| 0.1 mg/kg |
20 mL |
| 0.3-3 mg/kg |
100 mL |
Mix by gentle inversion. Do not shake.
Dilution for children:
Withdraw appropriate dose and required volume from the vial(s) and then dilute with NaCl 0.9% inj, as follows:
| Weight (kg) |
Dosage |
Final volume (mL) or concentration (mg/mL) |
| ≥3-<10 |
0.03-0.1 mg/kg |
0.1 mg/mL |
| 0.3 mg/kg |
5 mL |
| 0.6 mg/kg |
10 mL |
| 1 mg/kg |
20 mL |
| 2-3 mg/kg |
50 mL |
| ≥10-<20 |
0.03 mg/kg |
0.1 mg/mL |
| 0.1 mg/kg |
5 mL |
| 0.3 mg/kg |
10 mL |
| 0.6 mg/kg |
20 mL |
| 1 mg/kg |
50 mL |
| 2 mg/kg |
75 mL |
| 3 mg/kg |
100 mL |
| ≥20 |
0.03 mg/kg |
5 mL |
| 0.1 mg/kg |
10 mL |
| 0.3 mg/kg |
20 mL |
| 0.6 mg/kg |
50 mL |
| 1 mg/kg |
100 mL |
| 2 mg/kg |
200 mL |
| 3 mg/kg |
250 mL |
All solutions should be mixed by gentle inversion. Do not shake. Recommendations may vary among countries and between individual products (refer to specific product guidelines).
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Neonate and children. Pregnancy and lactation.
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Significant: Infusion-associated reactions, including acute phase reactions; treatment-emergent antidrug antibodies (ADA); transient transaminase (e.g. ALT or AST) elevations.
Cardiac disorders: Palpitations, tachycardia.
Eye disorders: Ocular hyperaemia or discomfort, eye pruritus.
Gastrointestinal disorders: Nausea, abdominal pain or discomfort, vomiting, upper abdominal pain, diarrhoea.
General disorders and administration site conditions: Pyrexia, pain, chills, fatigue, asthenia, catheter site-related reaction (e.g. pain, pruritus, swelling).
Hepatobiliary disorders: Hepatic pain.
Investigations: Increased C-reactive protein or serum ferritin; increased body temperature.
Musculoskeletal and connective tissue disorders: Myalgia, bone pain, arthralgia, back pain.
Nervous system disorders: Headache.
Respiratory, thoracic and mediastinal disorders: Pharyngeal oedema or swelling, throat tightness, wheezing, larynx or throat irritation, dyspnoea.
Skin and subcutaneous tissue disorders: Urticaria, pruritus, rash, erythema, fixed eruption, papular rash, macular rash, maculopapular rash, erythematous rash, pruritic rash, morbilliform rash, papule, macule.
Vascular disorders: Hypotension, hot flush, flushing.
Potentially Fatal: Hypersensitivity reactions (e.g. anaphylaxis, angioedema).
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IV: Z (Not recommended unless the benefits outweigh the potential risks due to risk of foetal malformations caused by increased sphingomyelin metabolite levels.)
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Women of childbearing potential must use effective birth control methods during therapy and for at least 14 days after stopping the treatment. This drug may cause hypotension, if affected, do not drive or operate machinery.
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Evaluate pregnancy status before treatment initiation. Obtain ALT and AST levels within 1 month before treatment initiation and during therapy. Monitor for infusion-associated reactions during and for an appropriate period of time after infusion.
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Description:
Overview: Olipudase alfa is an exogenous source of acid sphingomyelinase (ASM) enzyme.
Mechanism of Action: Olipudase alfa degrades sphingomyelin to ceramide and phosphocoline.
Pharmacodynamics: In patients with acid sphingomyelinase deficiency (ASMD), olipudase alfa has exhibited a transient post-infusion increase in plasma ceramide levels after each administration. During the dose escalation phase, plasma ceramide levels were significantly increased as compared with baseline. With repeated administration, plasma ceramide levels gradually decreased, and the pre-infusion levels during the maintenance phase were generally lower than baseline. Additionally, plasma lysosphingomyelin levels decreased after repeated olipudase alfa administration.
Pharmacokinetics:
Distribution: Volume of distribution: 13 L.
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Intact vials: Store between 2-8°C. Reconstituted vials with sterile water for inj: Store at 2-8°C for up to 24 hours or at 20-25°C for up to 6 hours. Diluted solution with NaCl 0.9% solution: Store at 2-8°C for up to 24 hours or 20-25°C for up to 12 hours. Storage recommendations may vary among countries and between individual products (refer to specific product guidelines).
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A16AB25 - olipudase alfa ; Belongs to the class of enzymes. Used in the treatment of alimentary tract and metabolism problems.
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Brayfield A, Cadart C (eds). Olipudase Alfa. Martindale: The Complete Drug Reference [online]. London. Pharmaceutical Press. https://www.medicinescomplete.com. Accessed 09/10/2025. Joint Formulary Committee. Olipudase Alfa. British National Formulary [online]. London. BMJ Group and Pharmaceutical Press. https://www.medicinescomplete.com. Accessed 09/10/2025. Olipudase Alfa-rpcp. UpToDate Lexidrug, AHFS DI (Adult and Pediatric) Online. American Society of Health-System Pharmacists, Inc. Waltham, MA. UpToDate, Inc. https://online.lexi.com. Accessed 09/10/2025. Olipudase Alfa. Gold Standard Drug Database in ClinicalKey [online]. Elsevier Inc. https://www.clinicalkey.com. Accessed 09/10/2025. Olipudase Alfa. UpToDate Lexidrug, Lexi-Drugs Multinational Online. Waltham, MA. UpToDate, Inc. https://online.lexi.com. Accessed 09/10/2025. Olipudase Alfa. UpToDate Lexidrug, Pediatric and Neonatal Lexi-Drugs Online. Waltham, MA. UpToDate, Inc. https://online.lexi.com. Accessed 10/10/2025. Paediatric Formulary Committee. Olipudase Alfa. BNF for Children [online]. London. BMJ Group, Pharmaceutical Press, and RCPCH Publications. https://www.medicinescomplete.com. Accessed 10/10/2025. Xenpozyme 20 mg Powder for Concentrate for Solution for Infusion (Aventis Pharma Ltd). MHRA. https://products.mhra.gov.uk. Accessed 09/10/2025. Xenpozyme 20 mg Powder for Concentrate for Solution for Infusion (Sanofi-Aventis [Malaysia] Sdn. Bhd.). National Pharmaceutical Regulatory Agency - Ministry of Health Malaysia. https://www.npra.gov.my. Accessed 09/10/2025. Xenpozyme Injection, Powder, Lyophilized, for Solution (Genzyme Corporation). DailyMed. Source: U.S. National Library of Medicine. https://dailymed.nlm.nih.gov/dailymed. Accessed 09/10/2025.
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