United States - English - NLM (National Library of Medicine)

cotherix, inc. - miglustat (unii: adn3s497az) (miglustat - unii:adn3s497az) - miglustat 100 mg - 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). none. risk summary based on findings from animal reproduction studies, miglustat 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. in animal reproduction studies, miglustat was maternally toxic in rabbits at exposures near the expected human therapeutic dose and caused embryo-fetal tox

Australia - English - Department of Health (Therapeutic Goods Administration)

janssen-cilag pty ltd - miglustat, quantity: 100 mg - capsule, hard - excipient ingredients: magnesium stearate; povidone; sodium starch glycollate; titanium dioxide; propylene glycol; butan-1-ol; isopropyl alcohol; purified water; strong ammonia solution; ethanol absolute; iron oxide black; ethanol; shellac; sulfuric acid; potassium hydroxide; industrial methylated spirit; ammonia; sorbitan monolaurate; gelatin; sodium lauryl sulfate - zavesca is indicated for the oral treatment of patients with mild to moderate type 1 gaucher disease, for whom enzyme replacement therapy is not a therapeutic option. zavesca is indicated for the treatment of the progressive neurological manifestations in adult and paediatric patients with niemann-pick type c disease.

Australia - English - Department of Health (Therapeutic Goods Administration)

takeda pharmaceuticals australia pty ltd - velaglucerase alfa, quantity: 400 u - injection, powder for - excipient ingredients: citric acid monohydrate; sucrose; polysorbate 20; sodium citrate dihydrate - vpriv is indicated for long-term enzyme replacement therapy (ert) for paediatric and adult patients with type 1 gaucher disease.

United Kingdom - English - MHRA (Medicines & Healthcare Products Regulatory Agency)

sanofi - eliglustat tartrate - capsule - 84.4mg

United States - English - NLM (National Library of Medicine)

ani pharmaceuticals, inc. - miglustat (unii: adn3s497az) (miglustat - unii:adn3s497az) - miglustat 100 mg - 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). 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 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 recommended human dose. no adverse developmental outcomes were observed with administration of miglustat to pregnant rats at dose levels 6 times the recommended human dose (see data) . the estimated background risk of major birth defects and miscarriage for the indicated population is unknown. all pregnancies have a background risk of birth defect, loss, or other adverse outcomes. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. clinical considerations disease-associated maternal and embryo-fetal risk pregnancy may exacerbate existing type 1 gaucher disease symptoms or result in new disease manifestations. type 1 gaucher disease manifestations may lead to adverse pregnancy outcomes including, hepatosplenomegaly which can interfere with the normal growth of a pregnancy and thrombocytopenia which can lead to increased bleeding and possible hemorrhage. data animal data in female rats given miglustat by oral gavage at doses of 20, 60, 180 mg/kg/day beginning 14 days before mating and continuing through gestation day 17 (organogenesis), increased post implantation loss, decreased embryo-fetal survival and decreased fetal and pup weights were observed at doses ≥60 mg/kg/day (≥2 times the human therapeutic dose on a mg/m2 basis). miglustat was also administered to pregnant rats by oral gavage at doses of 20, 60, 180 mg/kg/day from gestation day 6 through lactation (postpartum day 20). delayed and prolonged parturition with decreased live births were observed at doses ≥60 mg/kg/day (≥2 times the human therapeutic dose on a mg/m2 basis). in pregnant rabbits given miglustat by oral gavage at doses of 15, 30, 45 mg/kg/day during gestation days 6-18 (organogenesis), maternal toxicity, including maternal deaths (all doses), reduced food consumption (30 and 45 mg/kg/day), and decreased body weight gain (15 and 30 mg/kg/day), was observed. the 15 mg/kg/day dose level was 0.97 times the human therapeutic dose on a mg/m2 basis. in a pre- and postnatal development study in rats, miglustat was administered by oral gavage at doses of 20, 60, 180 mg/kg/day from gestation day 6 through day 20 of lactation, decreased live births were observed in dams, as well as decreased body weight gain in the offspring at doses ≥60 mg/kg/day (≥2 times the human therapeutic dose on a mg/m2 basis). there was no effect on behavioral and learning assessments, sexual maturation or reproductive performance of the offspring at doses up to 180 mg/kg/day (about 6 times the human therapeutic dose on a mg/m2 basis). risk summary there are no available data on the presence of miglustat in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. based on the physical properties of miglustat, miglustat is likely to be present in breast milk. because of the potential for serious adverse reactions in breastfed infants, advise women that breastfeeding is not recommended. infertility findings from a small clinical study in seven healthy adult males who received miglustat for six weeks did not indicate effects on male fertility. studies in male rats have shown that miglustat decreased fertility but findings were reversible. studies in female rats have shown increased post-implantation loss and decreased embryo-fetal survival [see use in specific populations (8.1), nonclinical toxicology (13.1)] . the safety and effectiveness of miglustat in pediatric patients have not been established. in a combined clinical trial safety data set of 45 patients less than 18 years of age exposed to miglustat in indications other than type 1 gaucher disease, the median weight and height percentiles adjusted for age and gender decreased during the first year of treatment but then stabilized. the mean length of exposure in these studies ranged from 2 to 2.6 years; some pediatric patients were exposed for up to 4 years. however, the effect of miglustat on long-term gain in weight and height in pediatric patients is unclear. clinical studies of miglustat capsules did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently than younger patients. other reported clinical experience has not identified differences in responses between elderly and younger patients. in general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, and cardiac function and of concomitant disease or other drug therapy. miglustat is known to be substantially excreted by the kidney, and the risk of adverse reactions to this drug may be greater in patients with impaired renal function [see clinical pharmacology (12.3)] . in patients with mild renal impairment (adjusted creatinine clearance 50-70 ml/min/1.73m2 ), miglustat capsule administration should commence at a dose of 100 mg twice per day. in patients with moderate renal impairment (adjusted creatinine clearance of 30-50 ml/min/1.73m2 ), miglustat capsule administration should commence at a dose of 100 mg once a day. use of miglustat capsules in patients with severe renal impairment (creatinine clearance < 30 ml/min/1.73m2 ) is not recommended. since elderly patients are more likely to have decreased renal function, care should be taken in dose selection, and it may be useful to monitor renal function. the impact of hemodialysis on the disposition of miglustat has not been investigated.

Israel - English - Ministry of Health

sanofi israel ltd - eliglustat - hard capsule - eliglustat 84.4 mg - eliglustat - cerdelga is indicated for the long-term treatment of adult patients with gaucher disease type 1 (gd1), who are cyp2d6 poor metabolisers (pms), intermediate metabolisers (ims) or extensive metabolisers (ems).

Israel - English - Ministry of Health

mbi pharma ltd., israel - miglustat - hard capsule - miglustat 100 mg - miglustat - miglustat dipharma is indicated for the oral treatment of mild to moderate type i gaucher disease. miglustat dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. miglustat dipharma is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with niemann-pick type c disease.

South Africa - English - South African Health Products Regulatory Authority (SAHPRA)

sanofi-aventis south africa (pty) ltd - injection - see ingredients - each 10,0 ml liquid contains imiglucerase 400,0 units

United States - English - NLM (National Library of Medicine)

breckenridge pharmaceutical, inc. - miglustat (unii: adn3s497az) (miglustat - unii:adn3s497az) - miglustat capsules are 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 tw

Singapore - English - HSA (Health Sciences Authority)

pfizer private limited - taliglucerase alfa - injection, powder, lyophilized, for solution - taliglucerase alfa 200units/vial