Deepika vegiraju at 15:55, 8 January 2023

2023-01-08T15:55:17Z

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== '''What is Miglustat?''' ==

* Miglustat ('''Zavesca''') is '''a glucosylceramide synthase inhibitor''' used in the therapy of [[Gaucher disease type 1|type 1 Gaucher disease]].
* Zavesca is an N-alkylated imino sugar, a synthetic analog of D-glucose.

[[File:Miglustat.svg|thumb|Miglustat]]

[[File:Miglustat 2.svg|thumb|Miglustat 2]]

[[File:Miglustat-enzyme complex.png|thumb|Miglustat-enzyme complex]]

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== '''What are the uses of this medicine?''' ==

* Miglustat ('''Zavesca''') is used alone to treat adults with mild to moderate [[Gaucher disease type 1|type 1 Gaucher disease]].
* Zavesca is used only in people who cannot be treated with [[Enzyme replacement therapy|enzyme replacement therapy]].

== '''How does this medicine work?''' ==

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* Type 1 Gaucher disease is caused by a '''functional deficiency of [[glucocerebrosidase]]''', the '''enzyme that mediates the degradation of the glycosphingolipid glucosylceramide'''.
* Miglustat (me' gloo stat) is a small molecule '''inhibitor of glucosylceramide synthase''', the first and rate controlling step in the pathway of glycolipid synthesis.
* By inhibiting the pathway, lower levels of substrate are available, less is available for lysosomal degradation and '''less glycosylceramide accumulates'''.
* Miglustat was shown to decrease the intracellular accumulation of glycosylceramide, the major glycolipid that accumulates in Gaucher disease, in animal models of the genetic disease.
* In several randomized controlled trials, miglustat was shown to decrease spleen and liver volume and increase [[hemoglobin]] and [[platelet]] counts in patients with type 1 Gaucher disease.
* Miglustat was also able to maintain clinical benefit in patients who had been maintained on long term enzyme replacement therapy with glucocerebrosidase infusions (the lysosomal enzyme that is deficient in type 1 Gaucher disease).

== '''Who Should Not Use this medicine ?''' ==

* This medicine have no usage limitations.

== '''What drug interactions can this medicine cause?''' ==

* Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Be sure to mention any of the following:
* [[imiglucerase]] (Cerezyme)

== '''Is this medicine FDA approved?''' ==

* Miglustat was approved in the United States in 2003 as oral therapy of type 1 Gaucher Disease in patients who are not eligible for [[enzyme replacement therapy]].
* Miglustat has also been evaluated in other lysosomal enzyme deficiencies and is approved in Europe, but not the United States as therapy of Niemann–Pick disease, type C. Miglustat is available in tablets of 100 mg under the brand name '''Zavesca'''.

== '''How should this medicine be used?''' ==

'''Recommended dosage:'''
* 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.

* May reduce dosage to 100 mg once or twice a day in some patients due to [[tremor]] or [[diarrhea]].

'''Administration:'''
* Miglustat comes as a capsule to take by mouth.
* It is usually taken with or without food and with plenty of water up to three times a day. To help you remember to take miglustat, take it at around the same time(s) every day.
* Swallow the capsules whole; do not split, chew, or crush them.
* Miglustat controls Gaucher disease but does not cure it.
* Continue to take miglustat even if you feel well.
* Do not stop taking miglustat without talking to your doctor.

== '''What are the dosage forms and brand names of this medicine?''' ==

This medicine is available in fallowing doasage form:
* '''As Capsules:''' 100 mg

This medicine is available in fallowing brand namesː
* '''Zavesca'''

=='''What side effects can this medication cause?'''==

The most common side effects of this medicine 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

Zavesca may cause serious side effects including:
* [[peripheral neuropathy]]
* shaky movements
* [[Diarrhea]]
* Weight loss
* Low [[platelet]] count

==''' What special precautions should I follow?''' ==

* [[Peripheral neuropathy]] have been reported in 3% of Gaucher's patients treated with Zavesca.Perform baseline and follow-up neurological evaluations at 6-month intervals in all patients.
* Patients have reported [[tremor]] or exacerbation of existing tremor on treatment. Reduce dose to ameliorate tremor or discontinue treatment if tremor does not resolve within days of dose reduction.
* Diarrhea and weight loss were common in clinical studies of patients treated with Zavesca. Evaluate for underlying gastrointestinal disease in patients who do not respond to usual interventions (e.g. diet modification).
* Mild reductions in [[platelet]] counts without association with bleeding were observed in some patients. Monitoring of platelet counts is recommended.
* Advise patients to take the next Zavesca capsule at the next scheduled time if a dose is missed.
* Inform patients of the potential risks and benefits of Zavesca and of alternative modes of therapy.
* Advise women not to breastfeed if they are taking Zavesca.
* Miglustat may damage [[sperm]]. Men who are taking miglustat should use effective birth control during treatment and for 3 months afterward.
* It is not known if Zavesca can pass into your breast milk and may harm your baby . Do not breastfeed during treatment with Zavesca. Talk to your healthcare provider about the best way to feed your baby during treatment with Zavesca.

== '''What to do in case of emergency/overdose?''' ==

* {{overdose}}

== '''Can this medicine be used in pregnancy?''' ==

* Zavesca may harm your unborn baby.
* Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Zavesca.

=='''Can this medicine be used in children?'''==

* The safety and effectiveness of Zavesca in pediatric patients have not been established.

== '''What are the active and inactive ingredients in this medicine?''' ==

* '''Active ingredient:''' miglustat
* '''Inactive ingredients:''' sodium starch glycolate, povidone (K30), and magnesium stearate.
* '''The capsule shell contains:''' gelatin and titanium dioxide; the edible printing ink contains black iron oxide and shellac.

== '''Who manufactures and distributes this medicine?''' ==

'''Manufactured for:'''
* [[Actelion Pharmaceuticals Ltd.|Actelion Pharmaceuticals US, Inc.]],
* a Janssen Pharmaceutical Company
* Titusville, NJ , USA

== '''What should I know about storage and disposal of this medication?''' ==

* Store Zavesca at room temperature between 68°F to 77°F (20°C to 25°C).

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[[Category:Iminosugars]]
[[Category:Transferase inhibitors]]
[[Category:Orphan drugs]]
[[Category:Piperidines]]

Miglustat - Revision history

Deepika vegiraju at 15:55, 8 January 2023