What is ZAVESCA and how is it used?

ZAVESCA is a prescription medicine used alone to treat adults with mild to moderate type 1 Gaucher disease. ZAVESCA is used only in people who cannot be treated with enzyme replacement therapy.

It is not known if ZAVESCA is safe and effective in children under 18 years of age.

What are the possible side effects of ZAVESCA?

ZAVESCA may cause serious side effects including:

• Numbness, tingling, pain, or burning in your hands or feet (peripheral neuropathy). Call your healthcare provider right away if you get numbness, tingling, pain, or burning in your hands or feet.
• Your healthcare provider may test your nerves (neurological exam) before you start ZAVESCA and during treatment with ZAVESCA.
• New or worsening hand tremors (shaky movements). Tremors are common with ZAVESCA and may begin within the first month of starting treatment. Sometimes the tremors may go away between 1 to 3 months with continued treatment. Your healthcare provider may lower your dose or stop ZAVESCA if you develop new or worsening hand tremors. Call your healthcare provider right away if you get new hand tremors during treatment with ZAVESCA or if the hand tremors you already have get worse.
• Diarrhea is common with ZAVESCA and sometimes can be serious. Your healthcare provider may prescribe another medicine (anti-diarrheal) to treat diarrhea if it is a problem for you and may recommend changes to your diet, such as avoiding foods high in carbohydrates. Talk with your healthcare provider about your diet if you have diarrhea.
• Weight loss is common with ZAVESCA and sometimes can be serious. You may lose weight when you start treatment with ZAVESCA.
• Low platelet count is common with ZAVESCA and can be serious. Your healthcare provider may do blood tests to monitor your blood platelet count during treatment with ZAVESCA.

The most common side effects of ZAVESCA include:

• weight loss
• stomach pain
• gas
• nausea and vomiting
• headache, including migraine
• back pain
• constipation
• dry mouth
• heaviness in arms and legs
• memory loss
• unsteady walking
• leg cramps
• dizziness
• weakness
• vision problems
• muscle cramps
• loss of appetite
• indigestion
• numbness, tingling, pain, or burning of your skin
• stomach bloating
• stomach pain not related to food
• menstrual changes

These are not all of the possible side effects of ZAVESCA.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

DESCRIPTION

Zavesca (miglustat capsules, 100 mg) is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for the first step in the synthesis of most glycosphingolipids.

Zavesca is an N-alkylated imino sugar, a synthetic analog of D-glucose.

The chemical name for miglustat is 1,5-(butylimino)-1,5-dideoxy-D-glucitol with the chemical formula C₁₀H₂₁NO₄ and a molecular weight of 219.28.

Miglustat is a white to off-white crystalline solid and has a bitter taste. It is highly soluble in water (>1000 mg/mL as a free base).

Zavesca is supplied in hard gelatin capsules each containing 100 mg miglustat for oral administration. Each Zavesca 100 mg capsule also contains sodium starch glycollate, povidone (K30), and magnesium stearate. Ingredients in the capsule shell include gelatin and titanium dioxide, and the shells are printed with edible ink consisting of black iron oxide and shellac.

INDICATIONS

Type 1 Gaucher Disease

ZAVESCA is indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to allergy, hypersensitivity, or poor venous access).

DOSAGE AND ADMINISTRATION

Instructions For Administration

Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease.

The recommended dose for the treatment of adult patients with type 1 Gaucher disease is one 100 mg capsule administered orally three times a day at regular intervals. If a dose is missed, the next ZAVESCA capsule should be taken at the next scheduled time.

It may be necessary to reduce the dose to one 100 mg capsule once or twice a day in some patients due to adverse reactions, such as tremor or diarrhea.

Patients With Renal Insufficiency

In patients with mild renal impairment (adjusted creatinine clearance 50-70 mL/min/1.73 m²), initiate ZAVESCA treatment at a dose of 100 mg twice per day. In patients with moderate renal impairment (adjusted creatinine clearance of 30-50 mL/min/1.73 m²), initiate ZAVESCA treatment at a dose of one 100 mg capsule per day. ZAVESCA is not recommended for use in patients with severe renal impairment (creatinine clearance <30 mL/min/1.73 m²) [see Use In Specific Populations].

HOW SUPPLIED

Dosage Forms And Strengths

Capsules

100 mg of miglustat, white opaque hard gelatin capsules with “OGT 918” printed in black on the cap and “100” printed in black on the body.

Storage And Handling

ZAVESCA is supplied in hard gelatin capsules containing 100 mg of miglustat. ZAVESCA^® 100 mg capsules are white opaque with “OGT 918” printed in black on the cap and “100” printed in black on the body.

ZAVESCA^® 100 mg capsules are packed in blister cards. Six blister cards of 15 capsules are supplied in each carton.

NDC 66215-201-90: carton containing 90 capsules
NDC 66215-201-15: blister card containing 15 capsules

Storage

Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F), [See USP Controlled Room Temperature].

Keep out of reach of children.

SIDE EFFECTS

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]