Mucopolysaccharidosis type IVA (Morquio A Syndrome, MPS IVA)

Active Ingredient: Elosulfase alfa

Indication for Elosulfase alfa

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 years old or older)
Therapeutic intent: Curative procedure

Elosulfase alfa is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.

For this indication, competent medicine agencies globally authorize below treatments:

2 mg/kg of body weight administered once a week

For:

Dosage regimens

Intravenous, 2 milligrams elosulfase alfa per kilogram of body weight, once weekly.

Detailed description

The recommended dose of elosulfase alfa is 2 mg/kg of body weight administered once a week. The total volume of the infusion should be delivered over approximately 4 hours.

Because of the potential for hypersensitivity reactions with elosulfase alfa, patients should receive antihistamines with or without antipyretics 30 to 60 minutes prior to start of infusion.

Elderly patients (≥65 years old)

The safety and efficacy of elosulfase alfa in patients older than 65 years has not been established, and no alternative treatment regimen can be recommended in these patients. It is not known whether elderly patients respond differently from younger patients.

Dosage considerations

For intravenous infusion only.

Active ingredient

Elosulfase alfa

Elosulfase alfa is a recombinant form of human N-acetylgalactosamine-6-sulfatase (rhGALNS) used for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA). Elosulfase alfa is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S.

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