Due to a supply shortage of Cerezyme, treatment recommendations for patients with Gaucher’s disease have been issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA ) and published in the communiqué of the FAMHP dated 18/08/2009.
The situation seems to be gradually returning to normal at the manufacturing site of Cerezyme. The recommendations have therefore been updated.
In anticipation of a complete return to normal conditions expected during the first quarter of 2010, recommendations have been updated and are listed below.
- When medically possible infants, children and adolescents should receive Cerezyme at a reduced dose or at a reduced infusion frequency. No patients should be treated at a dose lower than 15 units per kilogram body weight every two weeks or alternative treatment should be considered.
- Adult patients at high risk for the development of severe, life-threatening disease progression or pregnant women with symptomatic Gaucher disease should also receive Cerezyme at a reduced dose or at a reduced infusion frequency.
No patients should be treated at a dose lower than 15 U/ kg every two weeks, or alternative treatment should be considered.
Patients with such high risk include patients with one or more of the following criteria: platelet count less than 20,000 per µl, thrombocytopenia and bleeding, symptomatic anemia, severe co-morbidity requiring imiglucerase treatment, such as a condition that puts a patient at risk for bleeding (for example cirrhosis, major surgery), a need for chemotherapy, lung disease caused by Gaucher cell infiltration, or new acute bone event during the last 12 months.
- In patients without a high-risk for severe, life-threatening disease progression, an alternative treatment should be considered or treatment should be interrupted.
- All patients should be monitored for changes in haemoglobin, platelets and chitotriosidase levels, as appropriate, at baseline and bimonthly thereafter. Adults who demonstrate exacerbation of disease while on dose reduction/dose interruption are at high risk for the development of progressive disease or complications and should reinitiate the original treatment with Cerezyme, or alternative treatment should be considered.
Reporting of side effects will continue as normal, with doctors recording the batch numbers of the medicines in each patient’s records.
These are temporary recommendations and do not change the currently SPC for Cerezyme.