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

camber pharmaceuticals, inc. - sapropterin dihydrochloride (unii: rg277lf5b3) (sapropterin - unii:egx657432i) - sapropterin dihydrochloride tablets are indicated to reduce blood phenylalanine (phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin-(bh4-) responsive phenylketonuria (pku). sapropterin dihydrochloride tablets are to be used in conjunction with a phe-restricted diet.  none.  risk summary available pregnancy registry data have not reported an association with sapropterin dihydrochloride and major birth defects, miscarriage, or adverse maternal or fetal outcomes when sapropterin dihydrochloride was used during pregnancy (see data) .  an embryo-fetal development study with sapropterin dihydrochloride in rats using oral doses up to 3 times the maximum recommended human dose (mrhd) given during the period of organogenesis showed no effects. in a rabbit study using oral administration of sapropterin dihydrochloride during the period of organogenesis, a rare defect, holoprosencephaly, was noted at 10 ti

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

camber pharmaceuticals, inc. - sapropterin dihydrochloride (unii: rg277lf5b3) (sapropterin - unii:egx657432i) - sapropterin dihydrochloride powder for oral solution is indicated to reduce blood phenylalanine (phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin-(bh4-) responsive phenylketonuria (pku). sapropterin dihydrochloride powder for oral solution is to be used in conjunction with a phe-restricted diet. none. risk summary available pregnancy registry data have not reported an association with sapropterin dihydrochloride and major birth defects, miscarriage, or adverse maternal or fetal outcomes when sapropterin dihydrochloride was used during pregnancy ( see data ).  an embryo-fetal development study with sapropterin dihydrochloride in rats using oral doses up to 3 times the maximum recommended human dose (mrhd) given during the period of organogenesis showed no effects. in a rabbit study using oral administration of sapropterin dihydrochloride during the period of organogenesis, a rare defect, holopro

European Union - English - EMA (European Medicines Agency)

dipharma arzneimittel gmbh - sapropterin dihydrochloride - phenylketonurias - other alimentary tract and metabolism products, - sapropterin dipharma is indicated for the treatment of hyperphenylalaninaemia (hpa) in adults and paediatric patients of all ages with phenylketonuria (pku) who have been shown to be responsive to such treatment.sapropterin dipharma is also indicated for the treatment of hyperphenylalaninaemia (hpa) in adults and paediatric patients of all ages with tetrahydrobiopterin (bh4) deficiency who have been shown to be responsive to such treatment.

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

northstar rxllc - sapropterin dihydrochloride (unii: rg277lf5b3) (sapropterin - unii:egx657432i) - sapropterin dihydrochloride tablets are indicated to reduce blood phenylalanine (phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin-(bh4-) responsive phenylketonuria (pku). sapropterin dihydrochloride tablets are to be used in conjunction with a phe-restricted diet.  none.  risk summary available pregnancy registry data have not reported an association with sapropterin dihydrochloride and major birth defects, miscarriage, or adverse maternal or fetal outcomes when sapropterin dihydrochloride was used during pregnancy (see data) .  an embryo-fetal development study with sapropterin dihydrochloride in rats using oral doses up to 3 times the maximum recommended human dose (mrhd) given during the period of organogenesis showed no effects. in a rabbit study using oral administration of sapropterin dihydrochloride during the period of organogenesis, a rare defect, holoprosencephaly, was noted at 10 times the mrhd.  all pregnancies have a background risk of major birth defects, pregnancy loss, or other adverse pregnancy 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. the estimated background risk of major birth defects and miscarriage in pregnant women with pku who maintain blood phenylalanine concentrations greater than 600 micromol/l during pregnancy is greater than the corresponding background risk for pregnant women without pku.  clinical considerations disease-associated maternal and/or embryo-fetal risk  uncontrolled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects. to reduce the risk of hyperphenylalaninemia-induced fetal adverse effects, blood phenylalanine concentrations should be maintained between 120 and 360 micromol/l during pregnancy and during the 3 months before conception [see dosage and administration 2.1)].  data  human data uncontrolled maternal pku  available data from the maternal phenylketonuria collaborative study on 468 pregnancies and 331 live births in pku-affected women demonstrated that uncontrolled phe levels above 600 micromol/l are associated with a very high incidence of neurological, cardiac, facial dysmorphism, and growth anomalies. control of blood phenylalanine during pregnancy is essential to reduce the incidence of phe-induced teratogenic effects.  pregnancy registry data  data from 62 live births reported 3 abnormalities at birth (one case each of microcephaly, cleft palate, and tongue tie). these outcomes were associated with phe levels greater than 360 micromol/l during pregnancy.  animal data  no effects on embryo-fetal development were observed in a reproduction study in rats using oral doses of up to 400 mg/kg per day sapropterin dihydrochloride (about 3 times the mrhd of 20 mg/kg per day, based on body surface area) administered during the period of organogenesis. however, in a rabbit reproduction study, oral administration of a maximum dose of 600 mg/kg per day (about 10 times the mrhd, based on body surface area) during the period of organogenesis was associated with a non-statistically significant increase in the incidence of holoprosencephaly in two high dose-treated litters (4 fetuses), compared to one control-treated litter (1 fetus). risk summary  there are insufficient data to assess the presence of sapropterin in human milk and no data on the effects on milk production. in postmarketing pregnancy registries, a total of 16 women from both registries were identified as breastfeeding for a mean of 3.5 months. no lactation-related safety concerns were reported in infants of mothers nursing during maternal treatment with sapropterin dihydrochloride. sapropterin is present in the milk of lactating rats following intravenous administration, but not following oral administration.  the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for sapropterin dihydrochloride and any potential adverse effects on the breastfed child from sapropterin dihydrochloride or from the underlying maternal condition.  pediatric patients with pku, ages 1 month to 16 years, have been treated with sapropterin dihydrochloride in clinical trials [see  clinical studies (14)]. the efficacy and safety of sapropterin dihydrochloride have not been established in neonates. the safety of sapropterin dihydrochloride has been established in children younger than 4 years in trials of 6 months duration and in children 4 years and older in trials of up to 3 years in length [see adverse reactions (6.1)].  in children aged 1 month and older, the efficacy of sapropterin dihydrochloride has been demonstrated in trials of 6 weeks or less in duration [see clinical studies (14)].  in a multicenter, open-label, single arm study, 57 patients aged 1 month to 6 years who were defined as sapropterin dihydrochloride responders after 4 weeks of sapropterin dihydrochloride treatment and phe dietary restriction were treated for 6 months with sapropterin dihydrochloride at 20 mg/kg per day. the effectiveness of sapropterin dihydrochloride alone on reduction of blood phe levels beyond 4 weeks could not be determined due to concurrent changes in dietary phe intake during the study. mean (±sd) blood phe values over time for patients aged 1 month to <2 years and 2 to <7 years are shown in figure 1. figure 1: mean blood phe level over time by age (years) (n=57) *error bars indicate 95% confidence interval. clinical studies of sapropterin dihydrochloride in patients with pku did not include patients aged 65 years and older. it is not known whether these patients respond differently than younger patients. 

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

dr. reddys laboratories inc. - sapropterin dihydrochloride (unii: rg277lf5b3) (sapropterin - unii:egx657432i) - sapropterin dihydrochloride tablets are indicated to reduce blood phenylalanine (phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin- (bh4-) responsive phenylketonuria (pku). sapropterin dihydrochloride is to be used in conjunction with a phe-restricted diet. none. pregnancy exposure registry there is a pregnancy exposure registry has been established that monitors pregnancy outcomes in women who are exposed to sapropterin dihydrochloride during pregnancy. for more information regarding the registry program call 1-800-983-4587. risk summary available pregnancy registry data  have not reported an  association with sapropterin dihydrochloride and major birth defects, miscarriage, or adverse maternal or fetal outcomes when sapropterin dihydrochloride was used during pregnancy (see data) . an embryo-fetal development study with sapropterin dihydrochloride in rats using oral doses up to 3 times the maximum recommended human dose (mrhd)

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

biomarin pharmaceutical inc. - sapropterin dihydrochloride (unii: rg277lf5b3) (sapropterin - unii:egx657432i) - sapropterin dihydrochloride 100 mg - kuvan® is indicated to reduce blood phenylalanine (phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin- (bh4-) responsive phenylketonuria (pku). kuvan is to be used in conjunction with a phe-restricted diet. none. pregnancy exposure registry there is a pregnancy exposure registry that monitors pregnancy outcomes in women who are exposed to kuvan during pregnancy. for more information regarding the registry program call 1-800-983-4587. risk summary available pregnancy registry data  have not reported an  association with kuvan and major birth defects, miscarriage, or adverse maternal or fetal outcomes when kuvan was used during pregnancy (see data) . an embryo-fetal development study with sapropterin dihydrochloride in rats using oral doses up to 3 times the maximum recommended human dose (mrhd) given during the period of organogenesis showed no effects. in a rabbit study using oral administration of sapropterin dihydrochloride dur

Canada - English - Health Canada

dr reddy's laboratories ltd - sapropterin dihydrochloride - powder for solution - 100mg - sapropterin dihydrochloride 100mg

Canada - English - Health Canada

dr reddy's laboratories ltd - sapropterin dihydrochloride - powder for solution - 500mg - sapropterin dihydrochloride 500mg

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

northstar rxllc - sapropterin dihydrochloride (unii: rg277lf5b3) (sapropterin - unii:egx657432i) - sapropterin dihydrochloride powder for oral solution is indicated to reduce blood phenylalanine (phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (hpa) due to tetrahydrobiopterin-(bh4-) responsive phenylketonuria (pku). sapropterin dihydrochloride powder for oral solution is to be used in conjunction with a phe-restricted diet. none. risk summary available pregnancy registry data have not reported an association with sapropterin dihydrochloride and major birth defects, miscarriage, or adverse maternal or fetal outcomes when sapropterin dihydrochloride was used during pregnancy ( see data ).  an embryo-fetal development study with sapropterin dihydrochloride in rats using oral doses up to 3 times the maximum recommended human dose (mrhd) given during the period of organogenesis showed no effects. in a rabbit study using oral administration of sapropterin dihydrochloride during the period of organogenesis, a rare defect, holoprosencephaly, was noted at 10 times the mrhd.  all pregnancies have a background risk of major birth defects, pregnancy loss, or other adverse pregnancy 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. the estimated background risk of major birth defects and miscarriage in pregnant women with pku who maintain blood phenylalanine concentrations greater than 600 micromol/l during pregnancy is greater than the corresponding background risk for pregnant women without pku.  clinical considerations   disease-associated maternal and/or embryo-fetal risk  uncontrolled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects. to reduce the risk of hyperphenylalaninemia-induced fetal adverse effects, blood phenylalanine concentrations should be maintained between 120 and 360 micromol/l during pregnancy and during the 3 months before conception [see dosage and administration (2.1)]. data  human data  uncontrolled maternal pku  available data from the maternal phenylketonuria collaborative study on 468 pregnancies and 331 live births in pku-affected women demonstrated that uncontrolled phe levels above 600 micromol/l are associated with a very high incidence of neurological, cardiac, facial dysmorphism, and growth anomalies. control of blood phenylalanine during pregnancy is essential to reduce the incidence of phe-induced teratogenic effects.  animal data  no effects on embryo-fetal development were observed in a reproduction study in rats using oral doses of up to 400 mg/kg per day sapropterin dihydrochloride (about 3 times the mrhd of 20 mg/kg per day, based on body surface area) administered during the period of organogenesis. however, in a rabbit reproduction study, oral administration of a maximum dose of 600 mg/kg per day (about 10 times the mrhd, based on body surface area) during the period of organogenesis was associated with a non-statistically significant increase in the incidence of holoprosencephaly in two high dose-treated litters (4 fetuses), compared to one control-treated litter (1 fetus). risk summary   there are insufficient data to assess the presence of sapropterin in human milk and no data on the effects on milk production. in postmarketing pregnancy registries, a total of 16 women from both registries were identified as breastfeeding for a mean of 3.5 months. no lactation-related safety concerns were reported in infants of mothers nursing during maternal treatment with sapropterin dihydrochloride. sapropterin is present in the milk of lactating rats following intravenous administration, but not following oral administration.  the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for sapropterin dihydrochloride and any potential adverse effects on the breastfed child from sapropterin dihydrochloride or from the underlying maternal condition.  pediatric patients with pku, ages 1 month to 16 years, have been treated with sapropterin dihydrochloride in clinical trials [see clinical studies (14)].  the efficacy and safety of sapropterin dihydrochloride have not been established in neonates. the safety of sapropterin dihydrochloride has been established in children younger than 4 years in trials of 6 months duration and in children 4 years and older in trials of up to 3 years in length [see adverse reactions (6.1)].  in children aged 1 month and older, the efficacy of sapropterin dihydrochloride has been demonstrated in trials of 6 weeks or less in duration [see clinical studies (14)].  in a multicenter, open-label, single arm study, 57 patients aged 1 month to 6 years who were defined as sapropterin dihydrochloride responders after 4 weeks of sapropterin dihydrochloride treatment and phe dietary restriction were treated for 6 months with sapropterin dihydrochloride at 20 mg/kg per day. the effectiveness of sapropterin dihydrochloride alone on reduction of blood phe levels beyond 4 weeks could not be determined due to concurrent changes in dietary phe intake during the study. mean (±sd) blood phe values over time for patients aged 1 month to <2 years and 2 to <7 years are shown in figure 1. figure 1: mean blood phe level over time by age (years) (n=57) *error bars indicate 95% confidence interval. clinical studies of sapropterin dihydrochloride in patients with pku did not include patients aged 65 years and older. it is not known whether these patients respond differently than younger patients.

New Zealand - English - Medsafe (Medicines Safety Authority)

te arai biofarma limited - sapropterin dihydrochloride 100mg equivalent to 76.8 mg sapropterin - soluble tablet - 100 mg - active: sapropterin dihydrochloride 100mg equivalent to 76.8 mg sapropterin excipient: ascorbic acid colloidal silicon dioxide copovidone crospovidone mannitol riboflavin sodium stearyl fumarate - indicated for the treatment of hyperphenylalaninemia (hpa) in sapropterin-responsive adult and paediatric patients with phenylketonuria (pku) or tetrahydrobiopterin (bh4) deficiency.