Xenpozyme (olipudase alfa-rpcp) (Olipudase Alfa INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION) 20 mg/5.1mL

Xenpozyme (olipudase alfa) is an FDA-approved enzyme replacement therapy developed by Sanofi Genzyme for treating Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick disease types A and B. This orphan drug represents a significant advancement in treating this rare lysosomal storage disorder. It is administered intravenously and falls within the neurology and genetic medicine therapeutic categories.

Strength

20 mg/5.1mL

Pack Size

1 VIAL in 1 CARTON (58468-0050-1) / 5.1 mL in 1 VIAL

Manufacturer / TM Owner

Sanofi Genzyme

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Indications & Clinical Uses

Xenpozyme is indicated for the treatment of Acid Sphingomyelinase Deficiency (ASMD), a rare autosomal recessive lysosomal storage disorder affecting patients with Niemann-Pick disease types A and B. The drug targets patients who have deficient or dysfunctional acid sphingomyelinase enzyme activity, leading to accumulation of sphingomyelin in various tissues including the liver, spleen, lungs, and central nervous system. Xenpozyme works as an enzyme replacement therapy, providing functional acid sphingomyelinase to reduce sphingomyelin accumulation and alleviate disease manifestations. The patient population includes both pediatric and adult patients with confirmed ASMD diagnosis. By restoring enzymatic activity, Xenpozyme helps reduce organomegaly, improve pulmonary function, and slow neurological progression in eligible patients, offering hope for this previously untreatable genetic condition.

Side Effects

Common adverse events associated with Xenpozyme include infusion-related reactions such as fever, chills, flushing, and headache. Patients may experience fatigue, nausea, and elevated liver enzymes. Serious adverse events can include anaphylaxis, severe infusion reactions, and potential immune responses. Monitoring requirements include regular assessment of infusion tolerance, liver function tests, and pulmonary function assessments. Patients should be observed during and after infusions for hypersensitivity reactions. Long-term monitoring of disease progression markers, organomegaly reduction, and neurological status is essential to evaluate therapeutic efficacy and detect emerging complications.

Precautions & Warnings

Xenpozyme is contraindicated in patients with known hypersensitivity to olipudase alfa or any excipients. Caution is warranted in patients with severe pulmonary disease or compromised cardiac function. Pregnancy category data should be reviewed with healthcare providers; animal studies have not demonstrated fetal harm, but human data are limited. Pediatric patients require careful dose adjustment and monitoring. Patients with hepatic or renal impairment should be evaluated individually, though specific dose adjustments are not established. Pre-medication with antihistamines and corticosteroids may be considered to mitigate infusion reactions. Immunosuppressive therapy may be necessary in patients developing anti-drug antibodies.

Avoid Interactions

Limited specific drug-drug interactions have been formally documented for olipudase alfa. However, concurrent use of immunosuppressive agents may be necessary to manage immune responses. Patients receiving other intravenous therapies should have adequate spacing between infusions. No significant food interactions are known. Caution should be exercised when administering other medications that may increase infusion reaction risk. Healthcare providers should review all concurrent medications and supplements with patients, as individual patient factors may influence tolerability and efficacy.

Frequently Asked Questions

Xenpozyme is used to treat Acid Sphingomyelinase Deficiency (ASMD), commonly known as Niemann-Pick disease types A and B. This rare genetic disorder causes deficiency of the acid sphingomyelinase enzyme, leading to harmful accumulation of sphingomyelin in organs and tissues. Xenpozyme provides enzyme replacement therapy to reduce this accumulation and alleviate disease symptoms.

Xenpozyme is administered as an intravenous infusion. The drug is given through a vein, typically in a clinical or hospital setting where patients can be monitored for infusion reactions. Treatment is ongoing, with regular infusions scheduled according to the physician's prescribed regimen and individual patient response.

Common side effects include infusion-related reactions such as fever, chills, flushing, headache, and fatigue. Patients may also experience nausea and elevated liver enzymes. Most infusion reactions are mild to moderate and manageable with appropriate pre-medication and monitoring during administration.

Xenpozyme is manufactured by Sanofi Genzyme, a subsidiary of Sanofi specializing in rare genetic diseases and cell therapy. Sanofi Genzyme is committed to developing innovative treatments for patients with rare and debilitating genetic disorders.

Yes, Xenpozyme is an FDA-approved orphan drug, designating it for the treatment of a rare disease affecting a small patient population. Orphan drug status provides regulatory incentives and support for development of treatments for rare conditions like ASMD. This designation reflects the significant unmet medical need in the Niemann-Pick disease community.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated: 17 Jun 2026

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