Zavasca milustat

ZAVESCA® is a glucosylceramide synthase inhibitor 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.

We remain dedicated to assisting the type 1 Gaucher disease community by:

  • Helping patients get started on therapy through our specialty pharmacy, Accredo®
  • Educating about financial assistance programs which may be available to eligible patients

IMPORTANT SAFETY INFORMATION

WARNING AND PRECAUTIONS

Peripheral Neuropathy

In clinical trials, cases of peripheral neuropathy have been reported in 3% of Gaucher's patients treated with ZAVESCA.

Tremor

Approximately 30% of patients taking ZAVESCA have reported tremor or exacerbation of existing tremor. These tremors were described as an exaggerated physiological tremor of the hands.

Diarrhea and Weight Loss

Reductions in Platelet Count

ADVERSE REACTIONS

The most common serious adverse reaction reported with ZAVESCA treatment in clinical trials was peripheral neuropathy.

The most common adverse reactions requiring intervention were diarrhea and tremor.

Adverse reactions in ≥5% of patients in two open-label, uncontrolled monotherapy trials of ZAVESCA and in an open-label active controlled study (Study 1, Study 2, Study 3):

  • Diarrhea (89%, 89%, 100%)
  • Weight loss (39%, 67%, 67%)
  • Abdominal pain (18%, 50%, 67%)
  • Flatulence (29%, 44%, 50%)
  • Nausea (14%, 22%, 8%)
  • Vomiting (4%, 11%, 0%)
  • Headache (21%, 22%, 0%)
  • Migraine (0%, 6%, 0%)
  • Tremor (11%, 11%, 17%)
  • Leg cramps (4%, 11%, 8%)
  • Dizziness (0%, 11%, 8%)
  • Generalized weakness (0%, 0%, 17%)
  • Visual disturbance (0%, 17%, 0%)
  • Thrombocytopenia (7%, 6%, 0%)
  • Muscle cramps (0%, 11%, 0%)
  • Back pain (0%, 0%, 8%)
  • Constipation (0%, 0%, 8%)
  • Dry mouth (0%, 0%, 8%)
  • Heaviness in limbs (0%, 0%, 8%)
  • Memory loss (0%, 0%, 8%)
  • Unsteady gait (0%, 0%, 8%)
  • Anorexia (7%, 0%, 0%)
  • Dyspepsia (7%, 0%, 0%)
  • Paresthesia (7%, 0%, 0%)
  • Bloating (0%, 6%, 0%)
  • Abdominal distention (0%, 0%, 8%)
  • Epigastric pain not food-related (0%, 6%, 0%)
  • Menstrual disorder (0%, 6%, 0%)

DOSAGE AND ADMINISTRATION

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

Patients with Renal Insufficiency

DRUG INTERACTIONS

Co-administration of ZAVESCA and imiglucerase may lead to increased clearance of imiglucerase.

USE IN SPECIFIC POPULATIONS

Pregnancy

Based on animal data, ZAVESCA may cause fetal harm.

Nursing Mothers

Discontinue drug or nursing based on importance of the drug to the mother.

Please see accompanying full Prescribing Information.

Accredo is a registered trademark of Accredo Health Group, Inc.