OLIPUDASE ALFA-RPCP

Other

Brand names: XENPOZYME

Intravenous

Mechanism of Action

12.1 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

See full prescribing information

Contraindications

• 4 CONTRAINDICATIONS None. None. ( 4 )

FDA Warnings

5 WARNINGS AND PRECAUTIONS Infusion-Associated Reactions (IARs): If severe IARs occur, discontinue XENPOZYME and initiate appropriate medical treatment
3 ) Risk of Fetal Malformations During Dosage Initiation or Escalation in Pregnancy: 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 malformations
Consider the risks and benefits of re-administering XENPOZYME following a severe hypersensitivity reaction (including anaphylaxis)
In the postmarketing setting, 24 hours after receiving XENPOZYME at a higher than recommended initial dose, a 2-year-old male patient with ASMD, experienced fever, respiratory distress, hypotension, and death [see Overdosage (10) ]

Known Side Effects

8 documented side effects by frequency

Headache

>10%

Fatigue

1-10%

Weight Gain

1-10%

Nausea

1-10%

Fever

1-10%

Condition Worsened

1-10%

Vomiting

1-10%

Cough

0.1-1%

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Formulations

Dosage Forms

Tablet

Route

Intravenous