Also found in: Wikipedia.


(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
Mentioned in ?
References in periodicals archive ?
Also, a highly statistically significant treatment difference was observed in another predefined subgroup analysis, requested by the European Medicines Agency (EMA), that compared arimoclomol to placebo control in patients receiving miglustat as a part of routine clinical care (p-value =0.0071).
and Dipharma Francis Sri announced the ANDA approval for Miglustat 100 mg capsules, the first generic version of Actelion's Zavesca.
Miglustat (ZavescaW, Actelion Pharmaceuticals Ltd, Switzerland), is a reversible inhibitor of glucosylceramide synthase, that has been shown to be effective in the treatment of progressive neurological manifestations in particularly among those with Late-infantile or Juvenile-onset disease, and has been used in that indication in Europe since 2010.
Carglumic acid formulation is the fourth Dipharma product approaching the market: Diterin (sapropterin dihydrochloride 100 mg tabs) has already been approved in South Korea and Russia for the treatment of hyperphenylalaninemia (HPA) due to phenylketonuria (PKU), Miglustat (miglustat 100 mg caps) was submitted in the USA through an abbreviated new drug application (ANDA) in 2016 for the treatment of Gaucher disease and Disanit[R] (nitisinone capsules) was submitted in EU in the last quarter of 2016 for the treatment of hereditary tyrosinemia type I.
Recent data show that inhibition of GBA2 with miglustat reduces the inflammatory response in CF bronchial epithelial cells infected by P.
Improved neuroprotection using miglustat, curcumin and ibuprofen as a triple combination therapy in Niemann-Pick disease type c1 mice.
Substrate reduction therapy (miglustat, Zavesca[R]; Actelion Pharmaceuticals, Allschwill, Switzerland) has more recently been licensed for type 1 of Gaucher disease in adult patients with mild to moderate disease for whom enzyme replacement therapy with imiglucerase is not a therapeutic option (9-11).
An oral drug called miglustat worked in mice, but not in men.
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.