Zavesca (Miglustat)

The following serious adverse reactions are described below and elsewhere in the labeling:

• Peripheral Neuropathy [see WARNINGS AND PRECAUTIONS]
• Tremor [see WARNINGS AND PRECAUTIONS]
• Diarrhea and weight loss [see WARNINGS AND PRECAUTIONS]
• Reductions in platelet count [see WARNINGS AND PRECAUTIONS]

Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

The data described below reflect exposure of 80 patients with type 1 Gaucher disease in two open-label, uncontrolled, monotherapy trials, one open-label, active-controlled trial, and two extensions, who received Zavesca at doses ranging from 50mg to 200 mg three times daily. Patients were aged 18 to 69 years at first treatment. The population was evenly distributed by gender.

The most common serious adverse reaction reported with Zavesca treatment in clinical trials was peripheral neuropathy [see WARNINGS AND PRECAUTIONS].

The most commonly reported adverse reactions in patients treated with Zavesca (occuring in ≥ 5%) that were considered related to Zavesca are shown in Tables 1 and 2. [see WARNINGS AND PRECAUTIONS].

The most common adverse reactions requiring intervention were diarrhea and tremor. [see WARNINGS AND PRECAUTIONS].

In two open-label, uncontrolled monotherapy trials, adult type 1 Gaucher disease patients were treated with Zavesca at a starting dose of 100 mg three times daily (dose range 100 to 200 mg three times daily) for up to 12 months in 28 patients [Study 1], or at a dose of 50 mg three times daily for up to 6 months in 18 patients [Study 2]. Table 1 below lists adverse reactions that occurred during the trials in ≥ 5% of patients.

Table 1: Adverse Reactions in ≥ 5% of Patients in Two Open-Label, Uncontrolled Monotherapy Trials of Zavesca

In an open-label, active-controlled study, 36 adult type 1 Gaucher disease patients were treated with Zavesca, imiglucerase, or Zavesca plus imiglucerase [Study 3] for up to 12 months. Table 2 lists adverse reactions that occurred during the trial in ≥ 5% of patients.

Table 2: Adverse Reactions in ≥ 5% of Patients in Open-Label Active Controlled Study