The safety and tolerability of ZAVESCA® have been evaluated in 80 adult type 1 Gaucher disease patients in two open-label uncontrolled and one open-label active controlled trials. All 80 patients in the combined dataset from the clinical studies reported at least one adverse event during their treatment period.

In two open-label, uncontrolled monotherapy trials in adult type 1 Gaucher disease patients treated with ZAVESCA® at a starting dose of 100 mg three times daily (dose range 100 - 200 mg three times daily) for 12 months in 28 patients [Study 1], or at a dose of 50 mg three times daily for 6 months in 18 patients [Study 2], gastrointestinal events were observed in more than 80% of patients either at the outset of treatment, or intermittently during treatment. Diarrhea was observed in approximately 85% of patients. Weight loss has been observed in up to 65% of patients. (see PRECAUTIONS,

General, Diarrhea and Weight Loss).

In the two open-label, uncontrolled monotherapy trials, the Adverse Reactions by WHO body system and preferred term occurring with an incidence of ≥ 5%, are presented in Table 9 below.

In an open-label, active-controlled study, 36 adult type 1 Gaucher disease patients were treated with ZAVESCA®, Cerezyme, or ZAVESCA® + Cerezyme (Study 3). Gastrointestinal adverse events and weight loss were commonly seen in patients exposed to ZAVESCA®. The Adverse Reactions by WHO body system and preferred term occurring with an incidence of ≥ 5%, are presented in Table 10 below.

While co-administration of ZAVESCA® appeared to increase the clearance of Cerezyme by 70%, these results are not conclusive because of the small number of subjects studied and because patients took variable doses of Cerezyme. Combination therapy with Cerezyme® (imiglucerase) and ZAVESCA® is not indicated (see CLINICAL PHARMACOLOGY, Drug Interactions).

Bookmark this page: