Some of the views expressed in the following notes on newly approved products should be regarded as preliminary, as there may have been limited published data at the time of publication, and little experience in Australia of their safety or efficacy. However, the Editorial Executive Committee believes that comments made in good faith at an early stage may still be of value. Before new drugs are prescribed, the Committee believes it is important that more detailed information is obtained from the manufacturer's approved product information, a drug information centre or some other appropriate source.
Ceredase (Arrow Scientific/Genzyme)
vials containing 10 IU/mL and 80 IU/mL for intravenous infusion
Indication: Gaucher's disease type I
Gaucher's disease is an autosomal recessive lysosomal storage disease. There is a deficiency of glucocerebrosidase which can lead to an accumulation of the enzyme's substrate. Some patients may have a normal life, but others can develop problems which require long term enzyme replacement therapy. This product has approval for use in patients with significant hepatomegaly or splenomegaly, moderate to severe anaemia, thrombocytopenia with a bleeding tendency, and bone disease.
Alglucerase is a modified form of glucocerebrosidase derived from human placental tissue. The product contains some human chorionic gonadotrophin and there is the potential risk of a virus surviving the manufacturing process. Alglucerase is supplied in a solution containing human serum albumin. It is diluted before being infused over one or two hours.
Although the optimum use of the product is still being determined, it is effective when used for the recommended indications.