Miglustat

1 INDICATIONS AND USAGE

INDICATIONS & USAGE SECTION

2 DOSAGE AND ADMINISTRATION

DOSAGE & ADMINISTRATION SECTION

3 DOSAGE FORMS AND STRENGTHS

DOSAGE FORMS & STRENGTHS SECTION

Capsules: 100 mg of miglustat, white opaque hard gelatin capsules with "OGT 918" printed in black on the cap and "100" printed in black on the body.

4 CONTRAINDICATIONS

CONTRAINDICATIONS SECTION

None.

5 WARNINGS AND PRECAUTIONS

WARNINGS AND PRECAUTIONS SECTION

6 ADVERSE REACTIONS

ADVERSE REACTIONS SECTION

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) ]

7 DRUG INTERACTIONS

DRUG INTERACTIONS SECTION

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) ].

8 USE IN SPECIFIC POPULATIONS

USE IN SPECIFIC POPULATIONS SECTION

11 DESCRIPTION

DESCRIPTION SECTION

Miglustat (miglustat capsules, 100 mg) is a 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 solid and has a bitter taste. It is highly soluble in water (>1000 mg/mL as a free base). Miglustat is supplied in hard gelatin capsules each containing 100 mg miglustat for oral administration. Each miglustat 100 mg capsule also contains magnesium stearate, povidone (K30), and sodium starch glycolate. Ingredients in the capsule shell include gelatin and titanium dioxide, and the shells are printed with edible ink consisting of black iron oxide and shellac.

12 CLINICAL PHARMACOLOGY

CLINICAL PHARMACOLOGY SECTION

13 NONCLINICAL TOXICOLOGY

NONCLINICAL TOXICOLOGY SECTION

14 CLINICAL STUDIES

CLINICAL STUDIES SECTION

The efficacy of miglustat in type 1 Gaucher disease has been investigated in two open-label, uncontrolled trials and one randomized, open-label, active-controlled trial with enzyme replacement given as imiglucerase. Patients who received miglustat were treated with doses ranging from 100 to 600 mg a day, although the majority of patients were maintained on doses between 200 to 300 mg a day. Efficacy parameters included the evaluation of liver and spleen organ volume, hemoglobin concentration, and platelet count. A total of 80 patients were exposed to miglustat during the three trials and their extension period.

16 HOW SUPPLIED/STORAGE AND HANDLING

HOW SUPPLIED SECTION

Miglustat is supplied in hard gelatin capsules containing 100 mg miglustat. Miglustat 100 mg capsules are white opaque with "OGT 918" printed in black on the cap and "100" printed in black on the body. Miglustat 100 mg capsules are packed in blister cards. Six blister cards of 15 capsules are supplied in each carton. NDC 10148-201-90: carton containing 90 capsules NDC 10148-201-15: blister card containing 15 capsules

17 PATIENT COUNSELING INFORMATION

INFORMATION FOR PATIENTS SECTION

Advise the patient to read the FDA-approved patient labeling (Patient Information).

SPL UNCLASSIFIED SECTION

SPL UNCLASSIFIED SECTION

Manufactured for : CoTherix, Inc. Titusville, NJ 08560, USA JN20221206

SPL PATIENT PACKAGE INSERT SECTION

SPL PATIENT PACKAGE INSERT SECTION

PATIENT INFORMATION MIGLUSTAT (MIG-loo-stat) (miglustat) Capsules This Patient Information has been approved by the U.S. Food and Drug Administration. Revised: 12/2022 Read this Patient Information before you start taking miglustat and each time you get a refill. There may be new information. What is MIGLUSTAT? Miglustat is a prescription medicine used alone to treat adults with mild to moderate type 1 Gaucher disease. Miglustat is used only in people who cannot be treated with enzyme replacement therapy. It is not known if miglustat is safe and effective in children under 18 years of age. Before taking MIGLUSTAT, tell your healthcare provider about all of your medical conditions, including if you: have kidney problems are pregnant or plan to become pregnant. Miglustat may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with miglustat. are breastfeeding or plan to breastfeed. It is not known if miglustat can pass into your breast milk and may harm your baby. Do not breastfeed during treatment with miglustat. Talk to your healthcare provider about the best way to feed your baby during treatment with miglustat. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Miglustat may affect how other medicines work. How should I take MIGLUSTAT? Take miglustat exactly as your healthcare provider tells you to. Take miglustat at the same time each day. If you miss a dose of miglustat, skip that dose. Take the next miglustat capsule at the usual time. What are the possible side effects of MIGLUSTAT? Miglustat may cause serious side effects including: Numbness, tingling, pain, or burning in your hands or feet (peripheral neuropathy). Call your healthcare provider right away if you get numbness, tingling, pain, or burning in your hands or feet. Your healthcare provider may test your nerves (neurological exam) before you start miglustat and during treatment with miglustat. New or worsening hand tremors (shaky movements). Tremors are common with miglustat and may begin within the first month of starting treatment. Sometimes the tremors may go away between 1 to 3 months with continued treatment. Your healthcare provider may lower your dose or stop miglustat if you develop new or worsening hand tremors. Call your healthcare provider right away if you get new hand tremors during treatment with miglustat or if the hand tremors you already have get worse. Diarrhea is common with miglustat and sometimes can be serious. Your healthcare provider may prescribe another medicine (anti-diarrheal) to treat diarrhea if it is a problem for you and may recommend changes to your diet, such as avoiding foods high in carbohydrates. Talk with your healthcare provider about your diet if you have diarrhea. Weight loss is common with miglustat and sometimes can be serious. You may lose weight when you start treatment with miglustat. Low platelet count is common with miglustat and can be serious. Your healthcare provider may do blood tests to monitor your blood platelet count during treatment with miglustat. The most common side effects of miglustat 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 These are not all of the possible side effects of miglustat. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. How should I store MIGLUSTAT? Store miglustat at room temperature between 68°F to 77°F (20°C to 25°C). Keep miglustat and all medicines out of the reach of children. General information about the safe and effective use of MIGLUSTAT. Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use miglustat for a condition for which it was not prescribed. Do not give miglustat to other people, even if they have the same symptoms you have. It may harm them. You can ask your healthcare provider or pharmacist for information about miglustat that is written for health professionals. What are the ingredients in MIGLUSTAT? Active ingredient: miglustat Inactive ingredients: magnesium stearate, povidone (K30), and sodium starch glycolate. The capsule shell contains: gelatin and titanium dioxide; the edible printing ink contains black iron oxide and shellac. Manufactured for : CoTherix, Inc. Titusville, NJ 08560, USA JN20221206

PRINCIPAL DISPLAY PANEL - 100 mg Capsule Blister Card Carton

PACKAGE LABEL.PRINCIPAL DISPLAY PANEL

migLUstat capsules NDC 10148-201-90 100 mg This package is not child resistant. Contents: 90 Capsules (six blister cards of 15 capsules each) Oral use Rx only