Yargesa (miglustat)
Glucosylceramide Synthase (GCS) Inhibitors Oral Capsule
Last reviewed · Source: First Databank (FDB) drug data
Overview
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.
Miglustat, an N-alkylated imino sugar, is a competitive and reversible inhibitor of glucosylceramide synthase, the enzyme responsible for the formation of glucocerebroside, which accumulates in and causes clinical manifestations of Gaucher disease. Glucocerebroside is the substrate for glucocerebrosidase, the deficient lysosomal enzyme in patients with Gaucher disease. Miglustat acts as a substrate reduction therapy by inhibiting the formation and reducing accumulation of the substrate (glucocerebroside) for the deficient enzyme.
Because patients with type 1 Gaucher disease have some level of residual glucocerebrosidase activity, reduction in the amount of substrate allows the residual activity of the deficient enzyme to be more effective.
Administration with food decreases the rate but not the extent of miglustat absorption. Miglustat is excreted unchanged in urine, and clearance of the drug is reduced by approximately 40, 60, or at least 70% in patients with mild, moderate, or severe renal impairment, respectively. (See Dosage and Administration: Special Populations.)
USES: Miglustat is used for the long-term treatment of a certain rare genetic problem (Gaucher's disease). It works by decreasing the amount of a certain natural fatty substance (glucosylceramide) that builds up due to this condition. This can help improve symptoms of Gaucher's disease, such as tiredness, pain in the joints/bones, and a swollen liver or spleen.
Generic name: miglustat
Pronunciation: (mi-GLOO-stat)
Drug class: Glucosylceramide Synthase (GCS) Inhibitors
Therapeutic class: Metabolic Modifiers
Reference product & biosimilars
No reference, biosimilar or interchangeable associated with this drug.
Today’s intelligence on Yargesa
Indications
The following indications are listed for Yargesa:
- Gaucher's Disease
Also known as: Cerebroside Lipidosis, Familial Splenic Anemia, Type 1 Gaucher Disease.
Dosing
The recommended adult dosage of miglustat is 100 mg given at regular intervals 3 times daily. If a dose is missed, that dose should be skipped, and the next dose taken at the usual time. In some patients, it may be necessary to reduce the dosage to 100 mg once or twice daily because of adverse effects such as diarrhea or tremor.
Miglustat is administered orally.
| Drug label | Dosing type | Instructions |
|---|---|---|
| Yargesa 100 Mg Capsule | Maintenance | Take 1 capsule (100 mg) by oral route 3 times per day. |
| Drug label | Dosing type | Instructions |
|---|---|---|
| Miglustat 100 Mg Capsule | Maintenance | Take 1 capsule (100 mg) by oral route 3 times per day. |
Drug interactions
There is no enhanced messaging for this drug.
There are no contraindication interactions for this drug.
There are no severe interactions for this drug.
There are no moderate interactions for this drug.
Contraindications
None.
- Chronic kidney disease stage 4 (severe) GFR 15-29 ml/min
- Chronic kidney disease stage 5 (failure) GFr<15 ml/min
- Lactation
- 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
- Peripheral neuropathy
- Thrombocytopenic disorder
- Tremor
Adverse reactions
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.
Less Frequent
- Hypertension
- Peripheral neuropathy
Rare or Very Rare
- Thrombocytopenic disorder
More Frequent
- Acute abdominal pain
- Cramps
- Diarrhea
- Flatulence
- Headache disorder
- Nausea
- Weight loss
Less Frequent
- 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 or Very Rare
- Back pain
- Chills
- Flu-like symptoms
- Hypoesthesia
- Muscle weakness in limbs
- Pain
- Pain in extremities
- Unsteady gait
- Xerostomia
Precautions
No enhanced messaging available.
Safety and efficacy of miglustat have not been established in pediatric patients younger than 18 years of age.
Available data from postmarketing casereports with use of miglustat in pregnancy are insufficient to assess a drug-associatedrisk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Thereare risks associated with symptomatic Type I Gaucher disease in pregnancy, includinghepatosplenomegaly and thrombocytopenia. 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. Advise pregnant women of the potential risks to a fetus.
It is not known whether miglustat is distributed into human or animal milk; however, the drug is likely to be present in breastmilk. Thet effects of miglustat on the breastfed infant or on milk production are not known. Because of the potential for serious adverse reactions in nursing infants, breastfeeding during miglustat treatment is not recommended.
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.
ICD codes
| Code | Type | Description |
|---|---|---|
| Gaucher's disease | ||
| E75.22 | ICD10CM | Gaucher disease |
ICD code information from First Databank (FDB).
Formulary status
Search health-plan coverage for this drug by ZIP code.
Choose the first character of a health plan name.
Last reviewed · Source: First Databank (FDB) drug data
On this page
Actions
Sign in to save favorites
Saving this drug requires a free Physicians Office Resource account.