Molecule
Olipudase alfa is a prescription medicine indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. It is available as powder for injection for intravenous use. MedsAcross supplies Olipudase alfa for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.
Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review
From the regulator-approved product information. Each section names its source.
XENPOZYME 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.
Source: US FDA prescribing information
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.
Source: US FDA prescribing information
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.
Source: US FDA prescribing information
None. None.
Source: US FDA prescribing information
Boxed 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. 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.
Source: US FDA prescribing information
The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis Infusion-Associated Reactions (IARs) Elevated Transaminase Levels Most common adverse reactions in adult patients (incidence ≥10%) are headache, cough, diarrhea, hypotension, and ocular hyperemia. Most common adverse reactions in pediatric patients (incidence ≥20%) are pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis.
Source: US FDA prescribing information
Store refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze. For storage of reconstituted and diluted solution.
Source: US FDA prescribing information
For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.
2 presentations of Olipudase alfa, sorted by availability.
| Medicine | Manufacturer | Register | Availability |
|---|---|---|---|
| Xenpozyme 20 mg/5.1 mL powder for injectionIntravenous Cold chain | Genzyme | US FDA | Available on request |
| Xenpozyme 4 mg/1.1 mL powder for injectionIntravenous Cold chain | Genzyme | US FDA | Available on request |
1 brand names across 1 markets.
United States
Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.
Yes, on request. MedsAcross lists 2 presentations of Olipudase alfa and sources them for licensed hospitals, importers and physicians against valid import documents.
The catalogue lists 1 brand names for Olipudase alfa, including Xenpozyme.
Yes. The US FDA first approved Olipudase alfa on 31 August 2022, according to Drugs@FDA.
Yes. Olipudase alfa has a central EU marketing authorisation (status: authorised), according to European Medicines Agency data.
At least one listed presentation of Olipudase alfa must be kept cold, so it is shipped in temperature-controlled packaging. Check the storage line on each presentation.
Olipudase alfa is classified under ATC code A16AB25 (Olipudase alfa).
Tell us the strength, quantity and destination country. Our team replies with availability, lead time and the import documents needed.