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

pfizer laboratories div pfizer inc - taliglucerase alfa (unii: 0r4nlx88o4) (taliglucerase alfa - unii:0r4nlx88o4) - taliglucerase alfa 200 u in 5 ml - elelyso is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of type 1 gaucher disease. none. risk summary the limited available data on elelyso use in pregnant women are not sufficient to inform a drug-associated risk. however, there are clinical considerations [see clinical considerations] . in animal reproduction studies when pregnant rats and rabbits were administered taliglucerase alfa at intravenous doses up to 5 times the recommended human dose (rhd), there was no evidence of embryo-fetal toxicity [see data] . the estimated background risk of major birth defects and miscarriage for the indicated population(s) are unknown. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. clinical considerations disease-associated maternal and/or embryo/fetal risk women with type 1 gaucher disease have an increased risk of spontaneous abortion if diseas

Israel - English - Ministry of Health

pfizer pharmaceuticals israel ltd - taliglucerase alfa - powder for solution for infusion - taliglucerase alfa 200 u/vial - taliglucerase alfa - taliglucerase alfa - elelyso™ (taliglucerase alfa) for injection is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ert) for adults and pediatric patients with a confirmed diagnosis of type 1 gaucher disease

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

pfizer australia pty ltd - taliglucerase alfa, quantity: 212 u - injection, powder for - excipient ingredients: mannitol; citric acid; sodium citrate dihydrate; polysorbate 80 - elelyso is indicated for long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of type 1 gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anaemia, thrombocytopenia.

New Zealand - English - Medsafe (Medicines Safety Authority)

pfizer new zealand limited - taliglucerase alfa 200 u (200 u deliverable, plus 6% overage) - powder for injection - 200 u - active: taliglucerase alfa 200 u (200 u deliverable, plus 6% overage) excipient: citric acid mannitol polysorbate 80 sodium citrate dihydrate - elelyso is indicated for long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of type 1 gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anaemia, thrombocytopenia.

Canada - English - Health Canada

pfizer canada ulc - taliglucerase alfa - powder for solution - 200unit - taliglucerase alfa 200unit - enzymes

Singapore - English - HSA (Health Sciences Authority)

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

Canada - English - Health Canada

sanofi genzyme, a division of sanofi-aventis canada inc - eliglustat (eliglustat tartrate) - capsule - 84mg - eliglustat (eliglustat tartrate) 84mg - other miscellaneous therapeutic agents

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

genzyme corporation - eliglustat (unii: dr40j4wa67) (eliglustat - unii:dr40j4wa67) - eliglustat 84 mg - cerdelga is indicated for the long-term treatment of adult patients with gaucher disease type 1 (gd1) who are cyp2d6 extensive metabolizers (ems), intermediate metabolizers (ims), or poor metabolizers (pms) as detected by an fda-cleared test [see dosage and administration (2.1)] . limitations of use : - patients who are cyp2d6 ultra-rapid metabolizers (urms) may not achieve adequate concentrations of cerdelga to achieve a therapeutic effect [see clinical studies (14)] . - a specific dosage cannot be recommended for those patients whose cyp2d6 genotype cannot be determined (indeterminate metabolizers) [see clinical studies (14)] . cerdelga is contraindicated in the following patients based on cyp2d6 metabolizer status due to the risk of cardiac arrhythmias from prolongation of the pr, qtc, and/or qrs cardiac intervals. ems - taking a strong or moderate cyp2d6 inhibitor concomitantly with a strong or moderate cyp3a inhibitor [see drug interactions (7.1)] - moderate or severe hepatic impairment [see use in specific populations (8.7)] - mild hepatic impairment and taking a strong or moderate cyp2d6 inhibitor [see use in specific populations (8.7)] ims - taking a strong or moderate cyp2d6 inhibitor concomitantly with a strong or moderate cyp3a inhibitor [see drug interactions (7.1)] - taking a strong cyp3a inhibitor [see drug interactions (7.1)] - any degree of hepatic impairment [see use in specific populations (8.7)] pms - taking a strong cyp3a inhibitor [see drug interactions (7.1)] - any degree of hepatic impairment [see use in specific populations (8.7)] risk summary available data on cerdelga use in pregnant women includes 20 pregnancies that occurred during the clinical development program and a small number of post-marketing case reports. these data are not sufficient to assess drug-associated risks major birth defects, miscarriage, or adverse maternal or fetal outcomes. in animal reproduction studies in pregnant rats administered oral eliglustat during organogenesis, a spectrum of various developmental abnormalities were observed at doses 6 times the recommended human dose. no adverse developmental outcomes were observed with oral administration of eliglustat to pregnant rabbits at dose levels 10 times the recommended human dose [see data] . the estimated background risk of major birth defects and miscarriage in 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% to 4% and 15% to 20%, respectively. clinical considerations disease-associated maternal and/or embryo/fetal risk women with gaucher disease type 1 have an increased risk of spontaneous abortion, especially if disease symptoms are not treated and controlled pre-conception and during a pregnancy. pregnancy may exacerbate existing gaucher disease type 1 symptoms or result in new disease manifestations. gaucher disease type 1 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 reproduction studies have been performed in pregnant rats at oral doses up to 120 mg/kg/day (about 6 times the recommended human dose based on body surface area) and in pregnant rabbits at oral doses up to 100 mg/kg/day (about 10 times the recommended human dose based on body surface area) following administration of eliglustat during the period of organogenesis (gestation days 6 to 17 in the rat and 6 to 18 in the rabbit). in rats, at 120 mg/kg/day (about 6 times the recommended human dose based on body surface area), eliglustat increased the number of late resorptions, dead fetuses and post implantation loss, reduced fetal body weight, and caused fetal cerebral variations (dilated cerebral ventricles), fetal skeletal variations (poor bone ossification) and fetal skeletal malformations (abnormal number of ribs or lumbar vertebra). eliglustat-related effects on fetal rats were observed in association with signs of maternal toxicity. eliglustat did not cause fetal harm in rabbits at oral doses up to 100 mg/kg/day (about 10 times the recommended human dose based on body surface area). mild maternal toxicity was observed at the 100 mg/kg/day dose. in a pre and postnatal development study in rats (dosed daily from gestation day 6 to postpartum day 21), eliglustat did not show any significant adverse effects on pre and postnatal development at doses up to 100 mg/kg/day (about 5 times the recommended human dose based on body surface area). risk summary there are no human data available on the presence of eliglustat in human milk, the effects on the breastfed infant, or the effects on milk production. eliglustat and its metabolites were present in the milk of lactating rats [see data] . when a drug is present in animal milk, it is likely that the drug will be present in human milk. the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for cerdelga and any potential adverse effects on the breastfed child from cerdelga or from the underlying maternal condition. data in a milk excretion study in the rat, a single oral dose of 30 mg/kg [14 c]-labeled eliglustat was administered to lactating female rats at day 11 postpartum. approximately 0.23% of the administered radioactivity was excreted into the milk within 24 hours of dose administration. the concentration in the milk at 24 hours post dose was 16.3-fold higher than the plasma concentration. safety and effectiveness in pediatric patients have not been established. clinical studies of cerdelga did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. clinical experience has not identified differences in responses between the elderly and younger patients. use cerdelga in patients with renal impairment based on the patient's cyp2d6 metabolizer status [see clinical pharmacology (12.3)] . ems - avoid cerdelga in patients with end-stage renal disease (esrd) (estimated creatinine clearance (eclcr) less than 15 ml/min not on dialysis or requiring dialysis). - no dosage adjustment is recommended in patients with mild, moderate, or severe renal impairment (eclcr at least 15 ml/min). ims and pms - avoid cerdelga in patients with any degree of renal impairment. use cerdelga in patients with hepatic impairment based on cyp2d6 metabolizer status and concomitant use of cyp2d6 or cyp3a inhibitors [see clinical pharmacology (12.3)] . ems - cerdelga is contraindicated in patients with [see contraindications (4)] : severe (child-pugh class c) hepatic impairment moderate (child-pugh class b) hepatic impairment mild (child-pugh class a) hepatic impairment taking a strong or moderate cyp2d6 inhibitor - severe (child-pugh class c) hepatic impairment - moderate (child-pugh class b) hepatic impairment - mild (child-pugh class a) hepatic impairment taking a strong or moderate cyp2d6 inhibitor - reduce dosage frequency of cerdelga 84 mg to once daily [see dosage and administration (2.3)] in patients with mild hepatic impairment taking: a weak cyp2d6 inhibitor a strong, moderate, or weak cyp3a inhibitor - a weak cyp2d6 inhibitor - a strong, moderate, or weak cyp3a inhibitor - no dosage adjustment is recommended in patients with mild hepatic impairment, unless otherwise specified above. ims and pms - cerdelga is contraindicated in patients with any degree of hepatic impairment [see contraindications (4)] .

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

takeda pharmaceuticals america, inc. - velaglucerase alfa (unii: 23hye36b0i) (velaglucerase alfa - unii:23hye36b0i) - velaglucerase alfa 2.5 mg in 1 ml - vpriv is indicated for long-term enzyme replacement therapy (ert) for patients with type 1 gaucher disease. none. risk summary available data on use of velaglucerase alfa in pregnant women includes more than 300 pregnancies reported from the pharmacovigilance database and published observational cohort studies, including the international gaucher disease registry. while available data cannot definitively establish or exclude the absence of a velaglucerase alfa associated risk during pregnancy, these data have not identified an association with use of velaglucerase alfa during pregnancy and major birth defects, miscarriage, or adverse maternal or fetal outcomes. in animal reproduction studies no fetal harm was observed in rats or rabbits when velaglucerase alfa was administered intravenously during organogenesis at doses with exposures up to 1.8 times and 4.3 times, respectively, the recommended human daily dose (see data) . the estimated background risk of major birth defects and miscarriage for the indicate

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.