Miglustat (zavesca): the first drug for Gaucher’s disease The first drug developed by ACTELION PHARMS, Inc. for the treatment of Gaucher’s disease type I caused by a functional defect in the glucosidase enzyme zavesca, was approved by the U.S. FDA in July 2003 under the trade name zavesca. Gaucher disease is a rare genetic metabolic disorder caused by a functional defect in the glucosidase enzyme ceruloplasmin. This enzyme is involved in the degradation of sphingolipid glucocerebrosides sphingomyelin in the body, and when the degradation of sphingolipid glucocerebrosides sphingomyelin is impaired, lysosomes from megalophils rich in this substance accumulate in the body and a wide range of pathological reactions occur, including severe anemia, thrombocytopenia, hepatosplenomegaly, osteonecrosis and osteomalacia. Enzyme replacement is often used in treatment, and Meglut is used in adult patients who cannot be treated with enzyme replacement. Meglut reduces the rate of intrathecal glycolipid biosynthesis and lowers the concentration of glucosidic sphingomyelin to a low level in the body, which helps to improve hepatosplenomegaly and low hematocrit and platelet count levels in patients with type I Gaucher disease. The recommended dose is 100 mg once, 3 times a day. If patients have adverse reactions such as diarrhea and tremor, the dose can be reduced to 1 or 2 times a day; for mildly impaired renal function (creatinine clearance of 50-70 ml/min/ 1.73m2) the recommended dose is 100 mg once, twice a day; for mildly impaired renal function (creatinine clearance of 50-70 ml/min/ 1.73m2), the recommended dose is 100 mg once, twice a day; for moderately impaired kidney function (creatinine clearance of 30-50 ml/min/1.73m2), the recommended dose is 100 mg once, once a day; for severely impaired kidney function, the drug should not be used. During the initial 1st month of application of the drug, adverse reactions such as tremor, diarrhea, gastrointestinal discomfort, visual impairment, weight loss, numbness, pain, back pain, fatigue, burning sensation in the hands and feet, heaviness in the limbs, thrombocytopenia, etc. are common; in men, it may affect the quality of sperm, so contraception should be used as much as possible during the use of the drug, and in female patients it is recommended not to get pregnant while taking it. The specifications of Meglumet capsule formulations are 100mg per capsule and 20 capsules per box.