Miglustat
Generic Name: miglustat
Brand Names:
Miglustat
11 DESCRIPTION Miglustat capsules, 100 mg are glucosylceramide synthase inhibitor, which is a glucosyl transferase enzyme responsible for the first step in the synthesis of most glycosphingolipids. Miglustat is an N-alkylated imino sugar, a synthetic analog of D-glucose. The chemical name for miglustat is 1,5-(butylimino)-1,5-dideoxy-D-glucitol with the chemical formula C 10 H 21 NO 4 and a molecular weight of 219.28. Miglustat is a white to off-white crystalline powder and has a bitter taste.
Overview
11 DESCRIPTION Miglustat capsules, 100 mg are glucosylceramide synthase inhibitor, which is a glucosyl transferase enzyme responsible for the first step in the synthesis of most glycosphingolipids. Miglustat is an N-alkylated imino sugar, a synthetic analog of D-glucose. The chemical name for miglustat is 1,5-(butylimino)-1,5-dideoxy-D-glucitol with the chemical formula C 10 H 21 NO 4 and a molecular weight of 219.28. Miglustat is a white to off-white crystalline powder and has a bitter taste.
Uses
1 INDICATIONS AND USAGE Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.( 1.1 ) 1.1 Type 1 Gaucher Disease Miglustat is indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to allergy, hypersensitivity, or poor venous access).
Dosage
2 DOSAGE AND ADMINISTRATION Recommended dosage is 100 mg administered orally three times a day at regular intervals ( 2.1 ). May reduce dosage to 100 mg once or twice a day in some patients due to tremor or diarrhea ( 2.1 ). Adjust in patients with renal impairment ( 2.2 ): Renal Impairment Adjusted Creatinine Clearance (in mL/min/1.73 m 2 ) Recommendations Mild 50 – 70 Start dose at 100 mg twice a day Moderate 30 – 50 Start dose at 100 mg once a day Severe <30 Use is not recommended 2.1 Instructions for Administration Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease. 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.
Side Effects
6 ADVERSE REACTIONS The following serious adverse reactions are described below and elsewhere in the labeling: Peripheral Neuropathy [see Warnings and Precautions ( 5.1 )] Tremor [see Warnings and Precautions ( 5.2 )] Diarrhea and Weight Loss [see Warnings and Precautions ( 5.3 )] Reductions in Platelet Count [see Warnings and Precautions ( 5.4 )] The most common adverse reactions (incidence ≥5%) are: diarrhea, weight loss, stomach pain, gas, nausea and vomiting headache including migraine, tremor, leg cramps, dizziness, weakness, vision problems, thrombocytopenia, muscle cramps, back pain, constipation, dry mouth, heaviness in arms and legs, memory loss, unsteady walking, anorexia, indigestion, paresthesia, stomach bloating, stomach pain not related to food, and menstrual changes ( 6.1 )....
Interactions
7 DRUG INTERACTIONS While co-administration of miglustat appeared to increase the clearance of imiglucerase by 70%, these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase [see Clinical Pharmacology ( 12.3 )]. Co-administration of miglustat and imiglucerase may lead to increased clearance of imiglucerase.
Warnings
5 WARNINGS AND PRECAUTIONS Peripheral neuropathy : Perform baseline and follow-up neurological evaluations at 6-month intervals in all patients ( 5.1 ). Tremor : Reduce dose to ameliorate tremor or discontinue treatment if tremor does not resolve within days of dose reduction ( 5.2 ). Diarrhea and weight loss : Evaluate for underlying gastrointestinal disease in patients who do not respond to usual interventions (e.g. diet modification) ( 5.3 ). Reductions in Platelet Count : Mild reductions in platelet counts without association with bleeding were observed in some patients. Monitoring of platelet counts is recommended ( 5.4 ). 5.1 Peripheral Neuropathy In clinical trials, cases of peripheral neuropathy have been reported in 3% of Gaucher's patients treated with miglustat. 4 CONTRAINDICATIONS None. None ( 4 ).
Pregnancy
8.1 Pregnancy Risk Summary Based on findings from animal reproduction studies, miglustat capsules may cause fetal harm when administered to a pregnant woman. Available data from postmarketing case reports with miglustat use in pregnancy are insufficient to assess a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. There are risks associated with symptomatic Type I Gaucher disease in pregnancy, including hepatosplenomegaly and thrombocytopenia (see Clinical Considerations). Advise pregnant women of the potential risks to the fetus.
Storage
16 HOW SUPPLIED/STORAGE AND HANDLING Miglustat capsules are supplied in hard gelatin capsules containing 100 mg of miglustat. Miglustat 100 mg capsules are white opaque cap imprinted "NAV" with black ink and white opaque body imprinted "104" with black ink filled with white to off white powder. Miglustat capsules 100 mg are packed in blister cards.
Frequently Asked Questions
What is Miglustat used for?▼
1 INDICATIONS AND USAGE Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.( 1.1 ) 1.1 Type 1 Gaucher Disease Miglustat is indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to allergy, hypersensitivity, or poor venous access).
What are the side effects of Miglustat?▼
6 ADVERSE REACTIONS The following serious adverse reactions are described below and elsewhere in the labeling: Peripheral Neuropathy [see Warnings and Precautions ( 5.1 )] Tremor [see Warnings and Precautions ( 5.2 )] Diarrhea and Weight Loss [see Warnings and Precautions ( 5.3 )] Reductions in Platelet Count [see Warnings and Precautions ( 5.4 )] The most common adverse reactions (incidence ≥5%) are: diarrhea, weight loss, stomach pain, gas, nausea and vomiting headache including migraine, tremor, leg cramps, dizziness, weakness, vision problems, thrombocytopenia, muscle cramps, back pain, constipation, dry mouth, heaviness in arms and legs, memory loss, unsteady walking, anorexia, indigestion, paresthesia, stomach bloating, stomach pain not related to food, and menstrual changes ( 6.1 )....
Can I take Miglustat during pregnancy?▼
8.1 Pregnancy Risk Summary Based on findings from animal reproduction studies, miglustat capsules may cause fetal harm when administered to a pregnant woman. Available data from postmarketing case reports with miglustat use in pregnancy are insufficient to assess a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. There are risks associated with symptomatic Type I Gaucher disease in pregnancy, including hepatosplenomegaly and thrombocytopenia (see Clinical Considerations). Advise pregnant women of the potential risks to the fetus.
What are the important warnings for Miglustat?▼
5 WARNINGS AND PRECAUTIONS Peripheral neuropathy : Perform baseline and follow-up neurological evaluations at 6-month intervals in all patients ( 5.1 ). Tremor : Reduce dose to ameliorate tremor or discontinue treatment if tremor does not resolve within days of dose reduction ( 5.2 ). Diarrhea and weight loss : Evaluate for underlying gastrointestinal disease in patients who do not respond to usual interventions (e.g. diet modification) ( 5.3 ). Reductions in Platelet Count : Mild reductions in platelet counts without association with bleeding were observed in some patients. Monitoring of platelet counts is recommended ( 5.4 ). 5.1 Peripheral Neuropathy In clinical trials, cases of peripheral neuropathy have been reported in 3% of Gaucher's patients treated with miglustat. 4 CONTRAINDICATIONS None. None ( 4 ).
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Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.