IDURSULFASE

Other

Brand names: ELAPRASE

Intravenous

Mechanism of Action

12.1 Mechanism of Action Hunter syndrome (Mucopolysaccharidosis II, MPS II) is an X-linked recessive disease caused by insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. This enzyme cleaves the terminal 2- O -sulfate moieties from the glycosaminoglycans (GAG) dermatan sulfate and heparan sulfate. Due to the missing or defective iduronate-2-sulfatase enzyme in patients with Hunter syndrome, GAG progressively accumulate in the lysosomes of a variety of cells, leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction. ELAPRASE is intended to provide exogenous enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to the M6P receptors on the cell surface, leading to cellular internalization of the enzyme, targeting to intracellular lysosomes and subsequent catabolism of accumulated GAG.

Indications

1 INDICATIONS AND USAGE ELAPRASE is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II

MPS II). ELAPRASE has been shown to improve walking capacity in patients 5 years and older. In patients 16 months to 5 years of age

no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome

however

MPS II). ELAPRASE has been shown to improve walking capacity in patients 5 years and older. In patients 16 months to 5 years of age

no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome

Contraindications

• 4 CONTRAINDICATIONS None. None. ( 4 )

FDA Warnings

5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Including Anaphylaxis : Ensure that personnel administering product are adequately trained in cardio-pulmonary resuscitative measures and have ready access to emergency medical services (EMS) ( 5
Risk of Hypersensitivity, Serious Adverse Reactions, and Antibody Development in Hunter Syndrome Patients with Severe Genetic Mutations : Hunter syndrome patients aged 7 years and younger with complete gene deletion, large gene rearrangement, nonsense, frameshift, or splice site mutations experienced a higher incidence of hypersensitivity reactions, serious adverse reactions, and anti-idursulfase antibody development ( 5
Risk of Acute Respiratory Complications : Patients with compromised respiratory function or acute febrile or respiratory illness may be at higher risk of life-threatening complications from hypersensitivity reactions
1 Hypersensitivity Reactions Including Anaphylaxis Serious hypersensitivity reactions, including anaphylaxis, have occurred during and up to 24 hours after infusion

Known Side Effects

5 documented side effects by frequency

Fever

>10%

Cough

1-10%

Shortness of Breath

1-10%

General Unwellness

0.1-1%

Upper Respiratory Infection

0.1-1%

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Formulations

Dosage Forms

Tablet

Route

Intravenous