Zavesca

Zavesca is an FDA-registered drug product whose active ingredient is MIGLUSTAT. It is labelled by Actelion Pharmaceuticals US, Inc. taken by oral and marketed as a human prescription drug product. Identifiers on file: NDC 66215-201, application NDA021348, label effective 20230420. It is one of 2 FDA drug label records sharing the active ingredient MIGLUSTAT. This page reproduces the FDA-approved label text so you can check what a product is, who makes it and how it is supplied before discussing it with a pharmacist or prescriber.

Across FDA labelling, products containing MIGLUSTAT are most often described in connection with: type gaucher, indicated monotherapy, treatment adult, mild moderate, enzyme replacement, therapeutic option, usage zavesca, glucosylceramide synthase. Wording differs between labels, and the full indications section on this page takes precedence when it is present.

1 INDICATIONS AND USAGE ZAVESCA 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 ZAVESCA 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).

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. If a dose is missed, the next ZAVESCA capsule should be taken at the next scheduled time. It may be necessary to reduce the dose to one 100 mg capsule once or twice a day in some patients due to adverse reactions, such as tremor or diarrhea. 2.2 Patients with Renal Insufficiency In patients with mild renal impairment (adjusted creatinine clearance 50–70 mL/min/1.73 m 2 ), initiate ZAVESCA treatment at a dose of 100 mg twice per day. In patients with moderate renal impairment (adjusted creatinine clearance of 30–50 mL/min/1.73 m 2 ), initiate ZAVESCA treatment at a dose of one 100 mg capsule per day. ZAVESCA is not recommended for use in patients with severe renal impairment (creatinine clearance <30 mL/min/1.73 m 2 ) [see Use in Specific Populations (8.6) ].

4 CONTRAINDICATIONS None. None ( 4 ).

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 ). To report SUSPECTED ADVERSE REACTIONS, contact Janssen at 1-800-526-7736 (1-800- JANSSEN) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The data described below reflect exposure of 80 patients with type 1 Gaucher disease in two open-label, uncontrolled, monotherapy trials, one open-label, active-controlled trial, and two extensions, who received ZAVESCA at doses ranging from 50 mg to 200 mg three times daily. Patients were aged 18 to 69 years at first treatment. The population was evenly distributed by gender. The most common serious adverse reaction reported with ZAVESCA treatment in clinical trials was peripheral neuropathy [see Warnings and Precautions (5.1) ]. The most commonly reported adverse reactions in patients treated with ZAVESCA (occurring in ≥5%) that were considered related to ZAVESCA are shown in Tables 1 and 2 [see Warnings and Precautions (5.2 , 5.3) ]. The most common adverse reactions requiring intervention were diarrhea and tremor [see Warnings and Precautions (5.2 , 5.3) ]. In two open-label, uncontrolled monotherapy trials, adult type 1 Gaucher disease patients were treated with ZAVESCA at a starting dose of 100 mg three times daily (dose range 100 to 200 mg three times daily) for up to 12 months in 28 patients [Study 1], or at a dose of 50 mg three times daily for up to 6 months in 18 patients [Study 2]. Table 1 below lists adverse reactions that occurred during the trials in ≥5% of patients. Table 1: Adverse Reactions in ≥5% of Patients in Two Open-Label, Uncontrolled Monotherapy Trials of ZAVESCA Incidence of adverse reactions Study 1 (starting dose 100 mg three times daily) Study 2 (50 mg three times daily) Patients entered in Study (n) 28 18 Body System - Preferred Term % of patients reporting % of patients reporting Gastrointestinal System Diarrhea 89 89 Flatulence 29 44 Abdominal Pain 18 50 Nausea 14 22 Vomiting 4 11 Bloating 0 6 Anorexia 7 0 Dyspepsia 7 0 Epigastric pain not food-related 0 6 Metabolic and Nutritional Disorders Weight Decrease 39 67 Central and Peripheral Nervous System Headache 21 22 Tremor 11 11 Dizziness 0 11 Leg cramps 4 11 Paresthesia 7 0 Migraine 0 6 Vision Disorders Visual Disturbance 0 17 Musculoskeletal Disorders Cramps 0 11 Platelet, Bleeding, and Clotting Disorders Thrombocytopenia 7 6 Reproductive disorders, female Menstrual disorder 0 6 In an open-label, active-controlled study, 36 adult type 1 Gaucher disease patients were treated with ZAVESCA, imiglucerase, or ZAVESCA plus imiglucerase [Study 3] for up to 12 months. Table 2 lists adverse reactions that occurred during the trial in ≥5% of patients. Table 2: Adverse Reactions in ≥5% of Patients in Open-Label Active Controlled Study Incidence of adverse reactions ZAVESCA alone Imiglucerase alone Patients entered in Study (n) 12 12 Body System - Preferred Term % of patients reporting % of patients reporting Gastrointestinal System Diarrhea 100 0 Abdominal Pain 67 0 Flatulence 50 0 Constipation 8 0 Nausea 8 0 Dry Mouth 8 0 Body as a Whole Pain 0 8 Generalized weakness 17 0 Abdominal distension 8 0 Back pain 8 0 Heaviness in limbs 8 0 Metabolic and Nutritional Disorders Weight Decrease 67 0 Central and Peripheral Nervous System Tremor 17 0 Dizziness 8 0 Leg cramps 8 0 Unsteady gait 8 0 Psychiatric disorders Memory loss 8 0

FDA lists 2 drug label records with MIGLUSTAT as the active ingredient. The most common dosage forms and the manufacturers with the most records are summarised below; strengths, pack sizes and inactive ingredients can still differ between apparently similar products.

What is Zavesca used for?
1 INDICATIONS AND USAGE ZAVESCA 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 ZAVESCA is indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom …

Who makes Zavesca?
Actelion Pharmaceuticals US, Inc. is the labeler of record for Zavesca. 2 FDA label records share the active ingredient MIGLUSTAT, so generic and branded versions may come from different companies; the manufacturer named on your pack is the one that matters for any enquiry.

What form does Zavesca come in?
administered by oral. Strengths and pack sizes vary by label, so compare the NDC on your pack with the identifier listed on this page.

Is Zavesca prescription or over-the-counter?
The FDA label for this product is recorded as human prescription drug. Marketing status can differ between strengths and countries, so check the dispensing rules where you live.

Are there other products containing MIGLUSTAT?
Yes. 2 FDA drug label records list MIGLUSTAT as the active ingredient. Products with the same active ingredient can usually be substituted only when a prescriber or pharmacist confirms it is appropriate.

Has Zavesca been recalled?
No FDA recall record in our database matches this product name or manufacturer. Recall data is updated periodically, so a very recent notice may not appear here yet.

Where does this information come from?
All content is reproduced from FDA Structured Product Labelling data via openFDA. It is provided for identification and reference only and is not medical advice.