caveat
Risk of hypersensitivity
During myositis injection, the patient has observed a life-threatening sheath response, including sheath activation.
Due to the potential for severe infusion reactions, appropriate medical support measures should be in place when managing myositis.
ESCRIPTION
MYOZYMEQY (alglucosidase alfa) by human acidAlpha-glucosidase(GAA) consists of the most dominant coding of the nine observed haplotypes of this gene. MYOZYME is produced in Chinese hamster ovary cell lines by recombinant DNA technology. Alglucosidase 18 alfa catalyzes the hydrolysis of the α-1,4- and α-1,6-glycosidic linkages of glycogen lysosomal glycogen by the original sugar.
Glucosidase alpha is a glycoprotein with a cohesive mass of 99,377 daltons for the peptide chain and a total mass of about 109,000 daltons, including carbohydrates. Alglucosidase alfa has a specific activity of 3 to 5 U / mg (one unit is defined as the amount of activity that causes 1 mole of synthetic substrate hydrolysis per minute under validated assay conditions). MYOZYME is suitable for intravenous infusion. It is provided as sterile, pyrogen free, white to off-white, optional cake or powder form for reconstruction with 0.3 mL sterile water for injection (USP). Each 50 g vial contains 52.5 mg a/glucosidase alpha, 210 mg mannitol, 0.5 mg polysorbate 80, 9.9 mg disodium hydrogen phosphate heptahydrate, 31.2 mg sodium dihydrogen phosphate monohydrate. After reconstitution as indicated, each vial contained 10.5 mL of reconstituted solution and a total extractable volume of 10 mL of 5.0 mg / mL glucosidase alpha. MYOZYME contains no preservatives; each vial is for single use only.
Clinical pharmacology
Action mechanism
Pompe diseaseType II glycogen storage disease, GSD II, type II glycogenosis, acid maltase deficiency) is a hereditary glycogen metabolism disorder caused by a loss or significant deficiency of the lysosomal enzyme GAA.
Pharmacokinetics
Pharmacokinetic assessment of alglucosidase alfa In 13 infants with Pompe disease ranging from 1 month to 7 months, they received 20 mg / kg (about 4 hours of infusion) or 40 mg / kg (about 6.5) ) MYOZYME is injected every 2 weeks. The measurement of glucosidase alpha plasma concentration is based on activity assays using artificial matrices. Systemic exposure is roughly proportional to the dose between the 20 and 40 mg / kg doses.
Indications and usage
MYOZYME (alglucosidase alfa) is indicated for patients with Pompe disease (GAA deficiency). Compared with 210 untreated historical controls, MYOZYME can improve the apnea-free survival rate in infants with Pompe disease, while MYOZYME has not been fully studied for other forms of Pompe disease to ensure safety and efficacy (see Clinical the study).
Contraindications
Nobody knows.
Precautions
general
Patients with acute underlying disease at the time of infusion of MYOZYME appear to be at risk of inputting a response. The clinical condition of the patient should be carefully considered before giving MYOZYME.
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