Opfolda (miglustat)
Glucosylceramide Synthase (GCS) Inhibitors Oral Capsule
Last reviewed · Source: First Databank (FDB) drug data
Overview
Miglustat, a synthetic analog of D-glucose, is an enzyme stabilizer.
Miglustat is an N-alkylated iminosugar, a synthetic analog of D-glucose, and is classified as an enzyme stabilizer. Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa-atga. Bound miglustat is dissociated from cipaglucosidase alfa-atga after it is internalized and transported into lysosomes.
Miglustat alone has no pharmacological activity in cleaving glycogen.
The maximum concentration (Cmax) and AUC of miglustat increase proportionally over a dosage range of 130 mg to 260 mg. The mean time to reach the maximum concentration of miglustat (Tmax) ranges from 2 to 3 hours. Co-administration of miglustat with food is predicted to result in delayed absorption and decreased Cmax in healthy subjects.
The terminal elimination half-life of miglustat is approximately 6 hours. No clinically significant differences in the pharmacokinetics of miglustat were observed based on age (18 to 74 years) and sex.
USES: Miglustat is used in combination with another medication (cipaglucosidase alfa) to treat a certain inherited condition (Pompe disease). Cipaglucosidase alfa works by breaking down a certain substance (glycogen) that builds up in the body due to this condition. Miglustat helps cipaglucosidase alfa stay active in the blood which helps it work better.
Miglustat must be used with cipaglucosidase alfa because it does not decrease glycogen levels when used alone.
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 Opfolda
Indications
The following indications are listed for Opfolda:
- Lysosomal Alpha-1,4-Glucosidase Deficiency
Also known as: Acid Maltase Deficiency, Glycogen Storage Disease Type 2, Glycogen Storage Disease Type Ii, Glycogenosis Type Ii, Pompe's Disease.
Dosing
No dosing information is available for this tab.
Miglustat is administered orally as capsules.
Miglustat must be used in combination with cipaglucosidase alfa-atga. Refer to the prescribing information for cipaglucosidase alfa-atga for dosage and administration recommendations. Miglustat and cipaglucosidase alfa combination therapy should be started 2 weeks after the last dose of enzyme replacement therapy.
Miglustat should be taken approximately 1 hour before IV administration of cipaglucosidase alfa-atga.
The capsules should be swallowed whole with an unsweetened beverage (e.g., water, tea or coffee with no cream, sugar, or sweeteners). Do not consume other beverages or food for at least 2 hours prior to and 2 hours after administration of miglustat.
If a dose of miglustat is missed, cipaglucosidase alfa-atga should not be administered and treatment should be rescheduled at least 24 hours after miglustat was last taken. If both miglustat and cipaglucosidase alfa-atga doses are missed, re-start treatment as soon as possible.
Store miglustat capsules at 20-25degreesC with excursions permitted between 15-30degreesC. Store in the original container to protect the drug from light.
| Drug label | Dosing type | Instructions |
|---|---|---|
| Opfolda 65 Mg Capsule | Maintenance | Take 4 capsules (260 mg) by oral route every 2 weeks with only unsweetened beverages at least 2 hours before and after food or drink. |
No dosing information is available for this tab.
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
*Pregnancy.
- Lactation
- Chronic kidney disease stage 3A (moderate) GFR 45-59 ml/min
- Chronic kidney disease stage 3B (moderate) GFR 30-44 ml/min
- Chronic kidney disease stage 4 (severe) GFR 15-29 ml/min
- Pregnancy
There are no moderate contraindications for this drug.
Adverse reactions
The most common adverse effects of miglustat (>=5% of patients) reported in clinical studies include headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.
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 effectiveness of miglustat in combination with cipaglucosidase alfa-atga in pediatric patients have not been established.
There is insufficient evidence with miglustat in pregnant women to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Based on findings from animal reproduction studies, miglustat in combination with cipaglucosidase alfa-atga may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, miglustat 25 mg/kg orally alone or in combination with IV cipaglucosidase alfa-atga was administered every other day to pregnant females during organogenesis (gestation day 7 through 19). Great vessel and cardiac malformations were increased in offspring of pregnant rabbits at 3- and 16-fold, respectively, the maximum recommended human dose (MHRD) of miglustat and cipaglucosidase alfa-atga based on plasma AUC exposure. A No Observed Adverse Effect Level (NOAEL) for the combination was not identified.
In a pre- and post-natal development study in rats, miglustat 60 mg/kg orally alone or in combination with IV cipaglucosidase alfa-atga was administered every other day to pregnant females during organogenesis (gestation days 8 through 18). Increases in both maternal and pup mortality were observed following maternal treatment with miglustat in combination with cipaglucosidase alfa-atga and with cipaglucosidase alfa-atga alone. A NOAEL for the combination was not identified.
There are no data on the presence of miglustat in human milk, the effects on the breast-fed child, or the effects on milk production. Miglustat is present in animal milk. Based on findings in animal studies, the use of miglustat in combination with cipaglucosidase alfa-atga may lead to serious adverse reactions in breast-fed infants. Advise females that breastfeeding is not recommended while receiving treatment with miglustat and cipaglucosidase alfa-atga.
The manufacturer makes no specific dosage recommendations for geriatric patients.
Clinical studies with miglustat did not include sufficient numbers of patients aged 65 years and older to determine whether they respond differently than younger patients. Of the total number of patients treated with miglustat in combination with cipaglucosidase alfa-atga in clinical trials for late-onset Pompe disease, 11% (17 patients) were 65 to 74 years of age and none were >=75 years of age.
ICD codes
| Code | Type | Description |
|---|---|---|
| Lysosomal alpha-1,4-glucosidase deficiency | ||
| E74.02 | ICD10CM | Pompe disease |
ICD code information from First Databank (FDB).
Formulary status
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Last reviewed · Source: First Databank (FDB) drug data
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