Included in the formulation
Included in the list (Order of the Government of the Russian Federation No. 2782-r of 30.12.2014):VED
АТХ:A.16.A.X.06 Miglustat
Pharmacodynamics:Inhibits the synthase of glucosylceramide, which leads to a decrease in the amount of substrate for Gaucher's disease and Niemann-Pick. As a result, in the case of Gaucher's disease of type 1, there is no accumulation of glucosylceramide in lysosomes, which leads to pathological changes in many organs. With Niemann-Pick disease, the amount of glycosphingolipids in glial cells and neurons decreases.
Pharmacokinetics:After oral administration, it is quickly and completely absorbed in the gastrointestinal tract. The maximum concentration in the blood plasma is reached after 2 hours. Does not bind to plasma proteins. Penetrates through the blood-brain and placental barrier, is found in breast milk.
Metabolism in the liver.
The half-life is 6-7 hours. Elimination by the kidneys, up to 80% unchanged.
Indications:It is used for the treatment of Gaucher's disease of type 1 with moderate and easy flow or inability to perform enzyme replacement therapy. Used for the progression of neurological symptoms in patients with Niemann-Pick disease.
IV.E70-E90.E75.2 Other sphingolypidosis
VI.G60-G64.G63.3 * Polyneuropathy in other endocrine diseases and metabolic disorders (E00-E07 +, E15-E16 +, E20-E34 +, E70-E89 +)
Contraindications:Severe renal and hepatic insufficiency, individual intolerance, children under 4 years old.
Carefully:Renal and hepatic insufficiency of mild severity, hypersensitivity.
Pregnancy and lactation:Recommendations for FDA - Category C. Contraindicated in pregnancy and lactation.
Dosing and Administration:Use in children
Contraindicated up to four years of age.
Up to 12 years, the dose is calculated depending on the body surface area.
Adults
Inside, 200 mg 3 times a day.
The highest daily dose: 200 mg.
The highest single dose: 600 mg.
Side effects:Central and peripheral nervous system: tremor, peripheral neuropathy, amnesia, headache, dizziness, depression, insomnia.
Hemopoietic system: thrombocytopenia.
Digestive system: anorexia, flatulence, diarrhea, nausea, vomiting, constipation, dyspepsia, weight loss.
Musculoskeletal system: myasthenia gravis, muscle spasms.
Reproductive system: decreased libido.
Allergic reactions.
Overdose:Cases of overdose are not described.
Treatment is symptomatic.
Interaction:Not described.
Special instructions:Monitoring the amount of peripheral blood platelets.
Men are recommended to use reliable methods of contraception during the administration of the drug and three months after the end of therapy.
It is advisable to use caution when driving vehicles and working with moving mechanisms.