Cerezyme is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions:

For injection: 400 units of imiglucerase as a white to off-white lyophilized powder in a single-dose vial for reconstitution.

None.

Imiglucerase is a hydrolytic lysosomal glucocerebrosidase-specific enzyme. It is an analogue of the human enzyme b-glucocerebrosidase (b-D-glucosyl-N-acylsphingosine glucohydrolase, E.C. 3.2.1.45), produced by recombinant DNA technology using mammalian cell culture (Chinese hamster ovary). Purified imiglucerase is a monomeric glycoprotein of 497 amino acids, containing 4 N-linked glycosylation sites (Mr=60,430). Imiglucerase differs from placental glucocerebrosidase by one amino acid at position 495, where histidine is substituted for arginine. The oligosaccharide chains at the glycosylation sites have been modified to terminate in mannose sugars. The modified carbohydrate structures on imiglucerase are somewhat different from those on placental glucocerebrosidase.

Cerezyme (imiglucerase) for injection is intended for intravenous use. It is supplied as a sterile, nonpyrogenic, white to off-white lyophilized powder for reconstitution with Sterile Water for Injection, USP. Each single-dose vial contains 424 units imiglucerase, mannitol (340 mg), polysorbate 80, NF (1.06 mg), and sodium citrates: disodium hydrogen citrate (36 mg) and trisodium citrate (104 mg).

An enzyme unit (U) is defined as the amount of enzyme that catalyzes the hydrolysis of 1 micromole of the synthetic substrate para-nitrophenyl-b-D-glucopyranoside (pNP-Glc) per minute at 37°C. Reconstituted solutions have a pH of approximately 6.1.

Study RC 91-0110 was a randomized, double-blind, active-controlled study of 30 patients (17 male and 13 female), aged 12 to 69 years (mean age of 38 years in the Cerezyme group and mean age of 28 years in the alglucerase group at baseline), with Gaucher disease type 1 and a hemoglobin of at least 1 g/dL below the lower age limit for age and sex. Patients were randomized 1:1 to receive either Cerezyme 60 units/kg every other week or alglucerase for 6 months. Primary efficacy parameters were an increase in hemoglobin concentration of at least 1 g/dL, increase in platelet count and decrease in spleen and liver volume at 6 months. Efficacy results are shown in Table 1.

Bone x-rays showed improvements in cortical thickness and lucencies in 7 of 11 Cerezyme treated patients.

In study RC 92-0501, twenty-nine patients continued treatment for total duration of 24 months. Patients were unblinded at 9 months and allowed to cross-over to Cerezyme treatment. At 24 months, mean increase in hemoglobin was 2.4 g/dL, mean increase in platelet count was 40 ×10

Cerezyme (imiglucerase) for injection, 400 units as a white to off-white lyophilized powder in a single-dose vial: NDC 58468-4663-1

NDC 58468-4663-1

Cerezyme

400 units per vial

For intravenous infusion after

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