Universal reference book for medicines
Name of the preparation: NAGLAZYM

Active substance: galsulfase

Type: The drug for the treatment of hereditary enzymatic insufficiency

Manufacturer: BIOMARIN EUROPE (United Kingdom) manufactured by CATALENT UK PACKAGING (United Kingdom)
Description of the active substance:
This information is a reference and it is not enough that the drug has been prescribed by a doctor ..

PHARMACHOLOGIC EFFECT
A remedy for enzyme replacement therapy, recombinant human N-acetylgalactosamine 4-sulfatase.

The accumulation of mucopolysaccharides is due to the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans.Mucopolysaccharidosis type 6 (Maroto-Lamy syndrome) is characterized by the absence or pronounced decrease in the enzyme N-acetylgalactosamine 4-sulfatase.Deficiency of the activity of this enzyme leads to the accumulation of the substrate of glucosaminoglycan, dermatan sulfate, throughout the body and causes widespread structural and functional disorders of cells, tissues and organs.

Halsulfase as an exogenous enzyme penetrates into the lysosomes and enhances the catabolism of glycosaminoglycan.
Cell capture and penetration into the lysosomes is most likely accomplished by binding the oligosaccharide chain of mannose-6-phosphate-terminating galsulfase to specific mannose-6-phosphate receptors.
PHARMACOKINETICS
Pharmacokinetic parameters for the administration of galsulfas at a dose of 1 mg / kg 1 time / week: C max - 0.8 μg / ml;
AUC - 2.3 μg x h -1 / ml; V d - 103 ml / kg;clearance - 7.2 ml / kg / min; T 1/2 - 9 min.
INDICATIONS
In the complex therapy of mucopolysaccharidosis type 6 (Maroto-Lamy syndrome).

DOSING MODE
It is used IV in the form of infusion at a dose of 1 mg / kg 1 time / week.

SIDE EFFECT
Reactions arising during the infusion: most often - hives on the face and neck, bronchospasm, respiratory distress, apnea.

Often: headache, fever, arthralgia, vomiting, upper respiratory tract infections, abdominal pain, diarrhea, ear pain, cough, otitis media, the appearance of anti-gulsulfase-IgG antibodies.

CONTRAINDICATIONS
Hypersensitivity to gulsulfus.

PREGNANCY AND LACTATION
Adequate and strictly controlled clinical studies of the safety of the use of galsulfas during pregnancy have not been conducted.
The application is possible only if the intended benefit to the mother exceeds the potential risk to the fetus and only in certain cases of extreme necessity.
It is not known whether the gastrose is excreted in human milk.
Use during lactation is possible only if the intended benefit to the mother exceeds the potential risk for the infant.
In experimental studies, no data were obtained on impaired fertility or hazard to the fetus when galsulfas was administered at a dose of 3 mg / kg / day in animals.

APPLICATION FOR CHILDREN
The safety of gelsulfs for children under 5 years old has not been investigated.

SPECIAL INSTRUCTIONS
Before the introduction of an infusion, it is recommended to perform a premedication with antihistamines.
However, it should be borne in mind that patients with type 6 mucopolysaccharidosis often experience sleep apnea. The use of antihistamines increases the risk of developing this condition.
The safety of gelsulfs for children under 5 years old has not been investigated.

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