European Union - English - EMA (European Medicines Agency)

mendelikabs europe ltd - nitisinone - tyrosinemias - other alimentary tract and metabolism products, - treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (ht 1) in combination with dietary restriction of tyrosine and phenylalanine.

Canada - English - Health Canada

mendelikabs inc - nitisinone - capsule - 2mg - nitisinone 2mg - other miscellaneous therapeutic agents

Canada - English - Health Canada

mendelikabs inc - nitisinone - capsule - 5mg - nitisinone 5mg - other miscellaneous therapeutic agents

Canada - English - Health Canada

mendelikabs inc - nitisinone - capsule - 10mg - nitisinone 10mg - other miscellaneous therapeutic agents

Canada - English - Health Canada

mendelikabs inc - nitisinone - capsule - 20mg - nitisinone 20mg - other miscellaneous therapeutic agents

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

par pharmaceutical, inc. - nitisinone (unii: k5bn214699) (nitisinone - unii:k5bn214699) - nitisinone capsules are indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (ht-1) in combination with dietary restriction of tyrosine and phenylalanine. none. risk summary limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. animal reproduction studies have been conducted for nitisinone. in these studies, nitisinone was administered to mice and rabbits during organogenesis with oral doses of nitisinone up to 20 and 8 times respectively, the recommended initial dose of 1 mg/kg/day. in mice, nitisinone caused incomplete skeletal ossification of fetal bones and decreased pup survival at doses 0.4 times the recommended initial dose, and increased gestational length at doses 4 times the recommended initial dose. in rabbits, nitisinone caused maternal toxicity and incomplete skeletal ossification of fetal bones at doses 1.6 times the recommended initial dose [see data]. the b

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

bryant ranch prepack - nitisinone (unii: k5bn214699) (nitisinone - unii:k5bn214699) - nitisinone capsules are indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (ht-1) in combination with dietary restriction of tyrosine and phenylalanine. none. risk summary limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. animal reproduction studies have been conducted for nitisinone. in these studies, nitisinone was administered to mice and rabbits during organogenesis with oral doses of nitisinone up to 20 and 8 times respectively, the recommended initial dose of 1 mg/kg/day. in mice, nitisinone caused incomplete skeletal ossification of fetal bones and decreased pup survival at doses 0.4 times the recommended initial dose, and increased gestational length at doses 4 times the recommended initial dose. in rabbits, nitisinone caused maternal toxicity and incomplete skeletal ossification of fetal bones at doses 1.6 times the recommended initial dose [see data]. the background risk of major birth defects and miscarriage for the indicated population 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. data animal data reproduction studies have been performed in mice at oral doses of about 0.4, 4 and 20 times the recommended initial dose (1 mg/kg/day) and in rabbits at oral doses of about 1.6, 4 and 8 times the recommended initial dose based on the body surface area. in mice, nitisinone has been shown to cause incomplete skeletal ossification of fetal bones at 0.4, 4 and 20 times the recommended initial dose, increased gestational length at 4 and 20 times the recommended initial dose, and decreased pup survival at 0.4 times the recommended initial dose based on the body surface area. in rabbits, nitisinone caused incomplete skeletal ossification of fetal bones at 1.6, 4 and 8 times the recommended initial dose based on the body surface area. risk summary there are no data on the presence of nitisinone in human milk, the effects on the breastfed infant, or the effects on milk production. data suggest that nitisinone is present in rat milk due to findings of ocular toxicity and lower body weight seen in drug naive nursing rat pups. the development and health benefits of breastfeeding should be considered along with the mother's clinical need for nitisinone and any potential adverse effects on the breastfed infant from nitisinone or from the underlying maternal condition. the safety and effectiveness of nitisinone have been established in pediatric patients for the treatment of ht-1 in combination with dietary restriction of tyrosine and phenylalanine. use of nitisinone in pediatric patients is supported by evidence from one open-label, uncontrolled clinical study conducted in 207 patients with ht-1 ages 0 to 22 years (median age 9 months) [see clinical studies (14)]. clinical studies of nitisinone did not include any subjects aged 65 and over. no pharmacokinetic studies of nitisinone have been performed in geriatric patients. in general, dose selection for an elderly patient should be cautious reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and concomitant disease or other drug therapy in this patient population.

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

bryant ranch prepack - nitisinone (unii: k5bn214699) (nitisinone - unii:k5bn214699) - nitisinone capsules are indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (ht-1) in combination with dietary restriction of tyrosine and phenylalanine. none. risk summary limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. animal reproduction studies have been conducted for nitisinone. in these studies, nitisinone was administered to mice and rabbits during organogenesis with oral doses of nitisinone up to 20 and 8 times respectively, the recommended initial dose of 1 mg/kg/day. in mice, nitisinone caused incomplete skeletal ossification of fetal bones and decreased pup survival at doses 0.4 times the recommended initial dose, and increased gestational length at doses 4 times the recommended initial dose. in rabbits, nitisinone caused maternal toxicity and incomplete skeletal ossification of fetal bones at doses 1.6 times the recommended initial dose [see data]. the background risk of major birth defects and miscarriage for the indicated population 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. data animal data reproduction studies have been performed in mice at oral doses of about 0.4, 4 and 20 times the recommended initial dose (1 mg/kg/day) and in rabbits at oral doses of about 1.6, 4 and 8 times the recommended initial dose based on the body surface area. in mice, nitisinone has been shown to cause incomplete skeletal ossification of fetal bones at 0.4, 4 and 20 times the recommended initial dose, increased gestational length at 4 and 20 times the recommended initial dose, and decreased pup survival at 0.4 times the recommended initial dose based on the body surface area. in rabbits, nitisinone caused incomplete skeletal ossification of fetal bones at 1.6, 4 and 8 times the recommended initial dose based on the body surface area. risk summary there are no data on the presence of nitisinone in human milk, the effects on the breastfed infant, or the effects on milk production. data suggest that nitisinone is present in rat milk due to findings of ocular toxicity and lower body weight seen in drug naive nursing rat pups. the development and health benefits of breastfeeding should be considered along with the mother's clinical need for nitisinone and any potential adverse effects on the breastfed infant from nitisinone or from the underlying maternal condition. the safety and effectiveness of nitisinone have been established in pediatric patients for the treatment of ht-1 in combination with dietary restriction of tyrosine and phenylalanine. use of nitisinone in pediatric patients is supported by evidence from one open-label, uncontrolled clinical study conducted in 207 patients with ht-1 ages 0 to 22 years (median age 9 months) [see clinical studies (14)]. clinical studies of nitisinone did not include any subjects aged 65 and over. no pharmacokinetic studies of nitisinone have been performed in geriatric patients. in general, dose selection for an elderly patient should be cautious reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and concomitant disease or other drug therapy in this patient population.

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

bryant ranch prepack - nitisinone (unii: k5bn214699) (nitisinone - unii:k5bn214699) - nitisinone capsules are indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (ht-1) in combination with dietary restriction of tyrosine and phenylalanine. none. risk summary limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. animal reproduction studies have been conducted for nitisinone. in these studies, nitisinone was administered to mice and rabbits during organogenesis with oral doses of nitisinone up to 20 and 8 times respectively, the recommended initial dose of 1 mg/kg/day. in mice, nitisinone caused incomplete skeletal ossification of fetal bones and decreased pup survival at doses 0.4 times the recommended initial dose, and increased gestational length at doses 4 times the recommended initial dose. in rabbits, nitisinone caused maternal toxicity and incomplete skeletal ossification of fetal bones at doses 1.6 times the recommended initial dose [see data]. the background risk of major birth defects and miscarriage for the indicated population 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. data animal data reproduction studies have been performed in mice at oral doses of about 0.4, 4 and 20 times the recommended initial dose (1 mg/kg/day) and in rabbits at oral doses of about 1.6, 4 and 8 times the recommended initial dose based on the body surface area. in mice, nitisinone has been shown to cause incomplete skeletal ossification of fetal bones at 0.4, 4 and 20 times the recommended initial dose, increased gestational length at 4 and 20 times the recommended initial dose, and decreased pup survival at 0.4 times the recommended initial dose based on the body surface area. in rabbits, nitisinone caused incomplete skeletal ossification of fetal bones at 1.6, 4 and 8 times the recommended initial dose based on the body surface area. risk summary there are no data on the presence of nitisinone in human milk, the effects on the breastfed infant, or the effects on milk production. data suggest that nitisinone is present in rat milk due to findings of ocular toxicity and lower body weight seen in drug naive nursing rat pups. the development and health benefits of breastfeeding should be considered along with the mother's clinical need for nitisinone and any potential adverse effects on the breastfed infant from nitisinone or from the underlying maternal condition. the safety and effectiveness of nitisinone have been established in pediatric patients for the treatment of ht-1 in combination with dietary restriction of tyrosine and phenylalanine. use of nitisinone in pediatric patients is supported by evidence from one open-label, uncontrolled clinical study conducted in 207 patients with ht-1 ages 0 to 22 years (median age 9 months) [see clinical studies (14)]. clinical studies of nitisinone did not include any subjects aged 65 and over. no pharmacokinetic studies of nitisinone have been performed in geriatric patients. in general, dose selection for an elderly patient should be cautious reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and concomitant disease or other drug therapy in this patient population.

Ireland - English - HPRA (Health Products Regulatory Authority)

dipharma b.v. - nitisinone - capsule, hard - 10 milligram(s) - nitisinone