Miglustat

Drug overview for MIGLUSTAT (miglustat):

Generic name: MIGLUSTAT (mi-GLOO-stat)
Drug class: Glucosylceramide Synthase (GCS) Inhibitors
Therapeutic class: Metabolic Modifiers

Miglustat is an inhibitor of glucosylceramide synthase (ceramide glucosyltransferase), the enzyme responsible for the formation of glucocerebroside (glucosylceramide), which accumulates in and causes clinical manifestations of Gaucher disease.

No enhanced Uses information available for this drug.

DRUG IMAGES

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The following indications for MIGLUSTAT (miglustat) have been approved by the FDA:

Indications:
Gaucher's disease

Professional Synonyms:
Cerebroside lipidosis
Familial splenic anemia
Type 1 Gaucher disease

Dosing information for MIGLUSTAT
DRUG LABEL	DOSING TYPE	DOSING INSTRUCTIONS
MIGLUSTAT 100 MG CAPSULE	Maintenance	Adults take 1 capsule (100 mg) by oral route 3 times per day

Generic dosing information for MIGLUSTAT
DRUG LABEL	DOSING TYPE	DOSING INSTRUCTIONS
MIGLUSTAT 100 MG CAPSULE	Maintenance	Adults take 1 capsule (100 mg) by oral route 3 times per day

The following contraindication information is available for MIGLUSTAT (miglustat):

Overview
Contraindicated
Severe
Moderate

Drug contraindication overview.

None.

There are 3 contraindications. Absolute contraindication.

Contraindication List
Chronic kidney disease stage 4 (severe) GFR 15-29 ml/min
Chronic kidney disease stage 5 (failure) GFr<15 ml/min
Lactation

There are 4 severe contraindications. Adequate patient monitoring is recommended for safer drug use.

Severe List
Chronic kidney disease stage 2 (mild) GFR 60-89 ml/min
Chronic kidney disease stage 3A (moderate) GFR 45-59 ml/min
Chronic kidney disease stage 3B (moderate) GFR 30-44 ml/min
Pregnancy

There are 3 moderate contraindications. Clinically significant contraindication, where the condition can be managed or treated before the drug may be given safely.

Moderate List
Peripheral neuropathy
Thrombocytopenic disorder
Tremor

The following adverse reaction information is available for MIGLUSTAT (miglustat):

Overview
Severe
Less Severe

Adverse reaction overview.

Adverse effects reported in at least 5% of patients receiving miglustat monotherapy in open-label clinical trials include diarrhea, flatulence, abdominal pain, abdominal distension with or without gas, nausea, vomiting, bloating, anorexia, dyspepsia, epigastric pain (not related to food), constipation, dry mouth, weight loss, headache, tremor, dizziness, unsteady gait, leg cramps, cramps, back pain, paresthesia, heaviness in limbs, generalized weakness, migraine, visual disturbance, memory loss, thrombocytopenia, and menstrual disorder.

There are 3 severe adverse reactions.

More Frequent	Less Frequent
None.	Hypertension Peripheral neuropathy

Rare/Very Rare
Thrombocytopenic disorder

There are 37 less severe adverse reactions.

More Frequent	Less Frequent
Acute abdominal pain Cramps Diarrhea Flatulence Headache disorder Nausea Weight loss	Abdominal distension Anorexia Arthralgia Constipation Cramps in legs Dizziness Drowsy Dyspepsia Flank pain Gait abnormality General weakness Injection site sequelae Malaise Memory impairment Menstrual disorder Migraine Myalgia Paresthesia Tremor Visual changes Vomiting

Rare/Very Rare
Back pain Chills Flu-like symptoms Hypoesthesia Muscle weakness in limbs Pain Pain in extremities Unsteady gait Xerostomia

The following precautions are available for MIGLUSTAT (miglustat):

Pediatric
Pregnant
Lactation
Geriatric

Safety and efficacy of miglustat have not been established in pediatric patients younger than 18 years of age.

Contraindicated

None

Severe Precaution

None

Management or Monitoring Precaution

None

Category C. (See Users Guide.) There are no adequate and well-controlled studies of miglustat in pregnant women. Animal studies suggest that the drug may cause fetal harm; decreased live births and fetal weights, and maternal deaths were observed in rats and rabbits. Miglustat should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus.

It is not known whether miglustat is distributed into milk. Because of the potential for serious adverse reactions in nursing infants, a decision should be made whether to discontinue nursing or the drug, taking into account the importance of the drug to the woman.

Clinical studies of miglustat did not include sufficient numbers of patients 65 years of age or older to determine whether they respond differently than younger adults. Other clinical experience has not identified differences in response between geriatric patients and younger adults.