YARGESA- miglustat capsule

Country: United States

Language: English

Source: NLM (National Library of Medicine)

Active ingredient:

MIGLUSTAT (UNII: ADN3S497AZ) (MIGLUSTAT - UNII:ADN3S497AZ)

Available from:

Edenbridge Pharmaceuticals LLC.

Administration route:

ORAL

Prescription type:

PRESCRIPTION DRUG

Therapeutic indications:

YARGESA 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).  None 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 capsules 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. In animal reproduction studies, miglustat was maternally toxic in rabbits at exposures near the expected human therapeutic dose and caused embryo-fetal toxicities in rats at doses twice the

Product summary:

YARGESA is supplied in hard gelatin capsules containing 100 mg miglustat. YARGESA 100 mg capsules are white opaque with “709” printed in black on the body. YARGESA are packed in blister cards. Six blister cards of 15 capsules are supplied in each carton. NDC 42799-709-01: carton containing 90 capsules. One blister card of 15 capsules is supplied in each carton. NDC 42799-709-15: carton containing 15 capsules. Storage: Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F) (see USP Controlled Room Temperature). Keep out of reach of children

Authorization status:

Abbreviated New Drug Application

Summary of Product characteristics

YARGESA - MIGLUSTAT CAPSULE
EDENBRIDGE PHARMACEUTICALS LLC.
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
YARGESA SAFELY AND EFFECTIVELY.
SEE FULL PRESCRIBING INFORMATION FOR YARGESA.
YARGESA (MIGLUSTAT) CAPSULES, FOR ORAL USE
INITIAL U.S. APPROVAL: 2003
INDICATIONS AND USAGE
YARGESA 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).
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 )
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
DOSAGE FORMS AND STRENGTHS
Capsules: 100 mg (3)
CONTRAINDICATIONS
None (4)
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)
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥ to 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, c