154248-97-2

Basic information

• Product Name: Imiglucerase
• Synonyms: Imiglucerase;Cerezyme;Unii-Q6U6J48bwy;Ceramidase, glucosyl- [495-histidine] (human placenta isoenzyme)
• CAS NO: 154248-97-2
• Molecular Weight: 0
• Mol File: 154248-97-2.mol
• Article Data: 0

Chemical Properties

• Appearance: /
• CAS DataBase Reference: Imiglucerase(CAS DataBase Reference)
• NIST Chemistry Reference: Imiglucerase(154248-97-2)
• EPA Substance Registry System: Imiglucerase(154248-97-2)

Safety Data

• Hazardous Substances Data: 154248-97-2(Hazardous Substances Data)

Usage

Description

Imiglucerase, a mannose-terminated form of human placental glucocerebrosidase produced by recombinant technology, is a therapeutic enzyme used for the treatment of type-1 Gaucher's disease. It catalyzes the hydrolysis of glucocerebroside, preventing the accumulation of this lipid in organs and tissues. The modification by removal of the terminal sugars results in strong binding of the exposed mannose to lectins expressed on the surface of macrophages, which greatly increases the amount of enzyme taken up by the cells. Imiglucerase has demonstrated unequivocal efficacy in clinical trials, leading to improved hemoglobin levels, platelet counts, and overall health in patients with type-1 Gaucher's disease.

Uses

Used in Enzyme Replacement Therapy:
Imiglucerase is used as an enzyme replacement therapy for the treatment of type-1 Gaucher's disease. It helps in the hydrolysis of glucocerebroside, preventing its accumulation in organs and tissues, which is the primary cause of the disease.
Used in Clinical Trials:
Imiglucerase has been utilized in 6-month clinical trials, where it demonstrated significant improvement in all patients with type-1 Gaucher's disease. The enzyme replacement therapy led to increased hemoglobin levels and platelet counts, decreased incidence of epistaxis and bruising, reduced spleen and liver sizes, and improved skeletal parameters.
Used in Pharmaceutical Industry:
Under the brand name Cerezyme (Genzyme), Imiglucerase is used in the pharmaceutical industry for the development and production of therapeutic treatments for type-1 Gaucher's disease. Its efficacy and well-tolerated nature make it a valuable asset in the management and treatment of this genetic disorder.

Originator

Genzyme (U.S.A.)

Clinical Use

Type 1 Gaucher disease is a hereditary condition occurringin about 1:40,000 individuals. It is characterized by a functionaldeficiency in β-glucocerebrosidase enzyme activityand the resulting accumulation of lipid glucocerebroside intissue macrophages, which become engorged and aretermed Gaucher cells. Gaucher cells typically accumulate inthe liver, spleen, and bone marrow and, occasionally, inlung, kidney, and intestine. Secondary hematological sequelaeinclude severe anemia and thrombocytopenia in additionto characteristic progressive hepatosplenomegaly. Skeletalcomplications are common and are frequently the most debilitatingand disabling feature of Gaucher disease. Possibleskeletal complications are osteonecrosis, osteopenia withsecondary pathological fractures, remodeling failure, osteosclerosis,and bone crises.Cerezyme (Imiglucerase) is a recombinant, macrophagetargetedvariant of human β-glucocerebrosidase, purified fromCHO cells. It catalyzes the hydrolysis of the glycolipid glucocerebrosideto glucose and ceramide following the normaldegradation pathway for membrane lipids.Cerezyme is supplied as a lyophilized powder for reconstitution.The powder should be stored at 2°C to 8°C untilused. The reconstituted product for IV infusion is stable for12 hours at room temperature.