Finished product reference
Olipudase Alfa
Injection, Powder, Lyophilized, for Solution · Parenteral · 4 mg
- List code
- 53427
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during XENPOZYME administration.
What it is and how it works
Mechanism of Action ASMD is a lysosomal storage disease that results from reduced activity of the enzyme acid sphingomyelinase (ASM), caused by pathogenic variants in the sphingomyelin phosphodiesterase 1 gene. ASM degrades sphingomyelin to ceramide and phosphocholine. The deficiency of ASM causes an intra-lysosomal accumulation of sphingomyelin (as well as cholesterol and other cell membrane lipids) in various tissues. XENPOZYME provides an exogenous source of ASM. XENPOZYME is not expected to cross the blood-brain barrier or modulate the CNS manifestations of ASMD.
Indications
is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. XENPOZYME is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.
Dosage and administration (summary)
See Full Prescribing Information for important recommendations prior to XENPOZYME treatment initiation. Adults: Recommended starting dose is 0.1 mg/kg administered as an intravenous infusion. Pediatrics: Recommended starting dose is 0.03 mg/kg administered as an intravenous infusion. See Full Prescribing Information for the recommended dose escalation and maintenance dosage, dosage modifications to reduce the risk of adverse reactions, and preparation and administration instructions.
Contraindications
None. None.
Warnings and precautions
Infusion-Associated Reactions (IARs): If severe IARs occur, discontinue XENPOZYME and initiate appropriate medical treatment. Elevated Transaminases: Assess ALT and AST within one month prior to initiation of XENPOZYME, within 72 hours prior to any infusion during dose escalation, or prior to the next scheduled XENPOZYME infusion upon resuming treatment following a missed dose.
Pregnancy
Pregnancy Risk Summary Based on findings from animal reproduction studies, XENPOZYME may cause embryo-fetal harm when administered to a pregnant female. XENPOZYME dosage initiation or escalation, at any time during pregnancy, is not recommended as it may lead to elevated sphingomyelin metabolite levels that may increase the risk of fetal malformati…
Storage and supply
Storage and Handling Store refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze. For storage of reconstituted and diluted solution .
Source: Olipudase Alfa-Rpcp (Xenpozyme) — US FDA prescribing information, 2025-12-11, DailyMed. Summarised and shortened; the full label governs. Iranian registration, indications and labelling may differ.
Reference information for professional sourcing and registration enquiries, not medical advice or a promotion. Drug information is condensed from US FDA prescribing information (public domain, via openFDA and DailyMed) and does not replace the product’s approved labelling in Iran. Availability, origin and documentation are confirmed per enquiry.
Tell us the quantity, the market and the timing.
Send the molecule or ingredient, the specification and the destination. We reply from Tehran with the questions, the documents and the next step.