(mi-gloo-stat) ,


(trade name)


Therapeutic: none assigned
Pharmacologic: enzyme inhibitors
Pregnancy Category: X


Mild to moderate type 1 Gaucher's disease, when enzyme replacement therapy is not an option.


Competitively and reversibly inhibits glucosylceramide synthase which is the initial step in the production of glycosphingolipids. The glycosphingolipid glucosylceramide accumulates in tissue in Gaucher's disease.

Therapeutic effects

Decreased production/accumulation of glycosphingolipid glucosylceramide with decreased tissue damage.


Absorption: Well absorbed following oral administration.
Distribution: Distributes into extravascular tissues.
Metabolism and Excretion: Not metabolized; excreted mostly unchanged in urine.
Half-life: 6–7 hr.

Time/action profile (blood levels)

POunknown2–2.5 hr8 hr


Contraindicated in: Hypersensitivity; Obstetric / Lactation: Pregnancy or lactation; Severe renal impairment (<30 mL/min).
Use Cautiously in: Mild to moderate renal impairment (dose alteration recommended if CCr <70 mL/min); Geriatric: Consider age related decrease in body mass, cardiac, renal and hepatic function, other chronic illnesses and concurrent drug therapies; Pediatric: Children <18 yr (safety not established).

Adverse Reactions/Side Effects

Central nervous system

  • headache (most frequent)


  • abdominal pain (most frequent)
  • diarrhea (most frequent)
  • flatulence (most frequent)
  • nausea (most frequent)
  • weight loss (most frequent)
  • anorexia
  • dyspepsia


  • ↓ male fertility


  • thrombocytopenia


  • weight loss (most frequent)


  • paresthesia
  • peripheral neuropathy
  • tremor


Drug-Drug interaction

↑ clearance of imiglucerase (should not be used concurrently).


Oral (Adults) 100 mg three time daily at regular intervals.

Renal Impairment

Oral (Adults) CCr 50–70 mL/min- 100 mg twice daily; CCr 30–50 mL/min- 100 mg once daily.


Capsules: 100 mg

Nursing implications

Nursing assessment

  • Neurological evaluations should be performed at baseline and every 6 mo during therapy. If symptoms of peripheral neuropathy (numbness, tingling) occur, discontinuation of therapy may be considered.
  • Assess for tremor. May begin during first month of therapy and may resolve between 1–3 mo of therapy; may require discontinuation of therapy.
  • Assess for diarrhea and weight loss. Advise patients with diarrhea to avoid high carbohydrate foods.
  • Lab Test Considerations: May cause thrombocytopenia.

Potential Nursing Diagnoses

Deficient knowledge, related to medication regimen (Patient/Family Teaching)


  • Oral: Administer 3 times daily at regular intervals. Capsules should be swallowed whole with water. May be administered without regard to food.

Patient/Family Teaching

  • Instruct patient to take miglustat as directed at the same time each day. If a dose is missed, skip the dose and take the next capsule at the usual time. Advise patient that sharing of this medication may be dangerous.
  • Advise patient to notify healthcare professional immediately if pregnancy is planned or suspected or if breastfeeding. Instruct male patients to maintain a reliable form of contraception during and for at least 3 mo after therapy.
  • Advise patient to consult health care professional prior to taking and Rx or OTC mediations or herbal products concurrently with miglustat.
  • Instruct patient to notify healthcare professional if numbness, pain, or burning in the hands and feet occur or if tremor develops or existing tremor worsens.

Evaluation/Desired Outcomes

  • Decreases in spleen and liver volumes in patients with Gaucher's disease.
Drug Guide, © 2015 Farlex and Partners
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References in periodicals archive ?
and Dipharma Francis Sri announced the ANDA approval for Miglustat 100 mg capsules, the first generic version of Actelion's Zavesca.
Actelion Acquisition: The acquisition gives JNJ five currently-marketed therapies (Opsumit, Tracleer, Uptravi, Veletri and Ventavis) for the treatment of pulmonary hypertension and two currently-marketed specialty products, Valchlor gel (cutaneous T-cell lymphoma) and Zavesca (Gaucher disease in the U.S./Nieman-Pick disease in Europe).
Further research showed the availability of a drug called Zavesca which has shown to slow the progression of the disease.
The medicine being used in the trial, called Zavesca, is not available on the NHS but could help slow down GM1, which progressively destroys nerve cells in the brain and spinal cord, and many children with the condition do not survive early childhood.
* Zavesca is the only disease-modifying therapy reducing the progression of clinically relevant neurological symptoms in patients with Niemann-Pick type C.
Kings tried to stop the re-opening of the trials, giving a sentence where it was specified the payment of a fine--"zavesca," "gloaba," "prada"--of the part that would appeal the decision.
Actelion has received a warning letter in which the FDA criticised that the Swiss company did not report about side effects and death cases among patients which were taking its drugs Tracleer, Ventavis and Zavesca.
Another Gaucher drug on the market is Swiss drugmaker Actelion Pharmaceuticals Ltd.'s Zavesca. U.S.-traded shares of Shire closed the week up $1.88, or 3%, at $59.52.
She relies on a revolutionary drug called Zavesca to keep her alive, which costs the NHS pounds 2,500 a month to provide.
An alternative form of therapy is substrate reduction; medication with miglustat (Zavesca) reduces the amount of glucocerebroside, allowing the patient's depleted residual glucocerebrosidase activity to cope with the reduced amount of substrate.
Sustained therapeutic effects of oral miglustat (Zavesca, Nbutyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

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