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Guidance to the Gaucher Community-Cerezyme Stakeholders Working Group June 24, 2009

Group 1: Most vulnerable patients

a. Infants, children and adolescents (less than or equal to 18 years old) and patients with type 3 Gaucher disease should continue receiving Cerezyme according to their current dose and frequency, without any interruptions.

b. Adult patients (>18 years old), with active disease progression (e.g., pulmonary hypertension, active skeletal disease [bone crisis and/or osteonecrosis within previous 90 days], severe thrombocytopenia, severe anemia, or other signs or symptoms which would place the patient at a high risk of developing complications following a dose reduction, as determined by the patient’s physician) should continue Cerezyme according to their current dose and frequency, without any interruptions.

c. Pregnant women who are currently receiving Cerezyme should continue according to their current dose and frequency, without any interruptions, assuming the indication and need are clear and after a careful risk/benefit analysis has been conducted.

d. Newly diagnosed patients who meet the criteria for Group 1 (based on [a] or [b] above) are recommended to initiate treatment with Cerezyme promptly.

Group 2: Less vulnerable patients

a. Adult patients (>18 years old) without clinical evidence of active disease progression should reduce their dose of Cerezyme by 50% of their current dose, but not lower than 15U/kg every 2 weeks (if currently receiving infusions every 2 weeks) or not lower than 30U/kg every 4 weeks (if currently receiving infusions every 4 weeks).

b. It is recommended that patients in Group 2 should have their hemoglobin level, platelet count, and chitotriosidase activity level (if available) measured at baseline and every 4 weeks during the period of temporary dose reduction.

c. If patients in this group show signs or symptoms of clinical worsening, it is recommended that the Cerezyme dose be adjusted back to the dose used prior to the dose reduction.

d. Newly diagnosed patients who do not meet the criteria for Group 1 should defer initiating treatment with Cerezyme until the period of Cerezyme shortage has ended.

General Guidance for All Patients

a. The recommendations should be implemented immediately and widely in order to conserve an adequate supply of Cerezyme for the most vulnerable patients.

b. The recommendations should be continued until notification by Genzyme that adequate supply has been restored.

c. At the end of this temporary period of Cerezyme shortage, it is recommended that all patients should resume their previously prescribed dosage regimen.

d. The recommendations may be subject to change if the guidance is not widely adopted or if Cerezyme production timelines need to be revised.

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