ZAVESCA^® Clinical Trials

Study Design

ZAVESCA^® has been studied in approximately 80 patients with type 1 Gaucher disease across three clinical trials.

The majority of patients were in the clinical trial for 6-12 months.

The studies looked at changes in the sizes of the liver and spleen as well as the concentration of hemoglobin (a protein in red blood cells that carries oxygen) and number of platelets.

Study Findings

In clinical studies, patients with mild to moderate type ¹ Gaucher disease taking ZAVESCA^® achieved*:

Reduced swelling of
liver and spleen

Increased levels of
hemoglobin

Increased levels of
platelets

*The results shown above varied among patients and clinical trials. Overall, patients taking ZAVESCA^® had decreases in liver and spleen volume. Changes in hemoglobin levels and platelet counts (both increases and decreases) were seen across the studies.

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 doctor right away if you get numbness, tingling, pain, or burning in your hands or feet. Your doctor 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 doctor may lower your dose or stop ZAVESCA^® if you develop new or worsening hand tremors. Call your doctor right away if you get new hand tremors while taking ZAVESCA^® or if the hand tremors you already have get worse.
• Diarrhea is common with ZAVESCA^® and sometimes can be serious. Your doctor 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 doctor 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 doctor may do blood tests to monitor your blood platelet count.

The most common side effects of ZAVESCA^® are:

Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of ZAVESCA^®. For more information, ask your doctor or pharmacist.

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

Please see full Prescribing Information and Patient Information.