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

novartis gene therapies, inc. - adeno-associated virus (unii: b769i4xpy3) (adeno-associated virus - unii:b769i4xpy3) - zolgensma is an adeno-associated virus (aav) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (sma) with bi-allelic mutations in the survival motor neuron 1 (smn1) gene. limitations of use - the safety and effectiveness of repeat administration of zolgensma have not been evaluated [see adverse reactions (6.2)] . - the use of zolgensma in patients with advanced sma (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see clinical studies (14)] . none. risk summary there are no available data regarding zolgensma use in pregnant women. no animal reproductive and developmental toxicity studies have been conducted with zolgensma. in the united states 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. risk summary there is no information available on the presence of zolgensma in human milk, the effects on the breastfed infant, or the effects on milk production. the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for zolgensma and any potential adverse effects on the breastfed child from zolgensma or from the underlying maternal condition. administration of zolgensma to premature neonates before reaching full-term gestational age is not recommended, because concomitant treatment with corticosteroids may adversely affect neurological development. delay zolgensma infusion until the corresponding full-term gestational age is reached. there is no information on whether breastfeeding should be restricted in mothers who may be seropositive for anti-aav9 antibodies. the safety of zolgensma was studied in pediatric patients who received zolgensma infusion at age 0.3 to 7.9 months (weight range, 3.0 kg to 8.4 kg) [see adverse reactions (6)] . the efficacy of zolgensma was studied in pediatric patients who received zolgensma infusion at age 0.5 to 7.9 months (weight range, 3.6 kg to 8.4 kg) [see clinical studies (14)] . zolgensma therapy should be carefully considered in patients with liver impairment. cases of acute serious liver injury and acute liver failure have been reported with zolgensma in patients with preexisting liver abnormalities. in clinical trials, elevation of aminotransferases was observed in patients following zolgensma infusion [see warnings and precautions (5.1)] .

European Union - English - EMA (European Medicines Agency)

novartis europharm limited  - onasemnogene abeparvovec - muscular atrophy, spinal - other drugs for disorders of the musculo-skeletal system - zolgensma is indicated for the treatment of:patients with 5q spinal muscular atrophy (sma) with a bi-allelic mutation in the smn1 gene and a clinical diagnosis of sma type 1, orpatients with 5q sma with a bi-allelic mutation in the smn1 gene and up to 3 copies of the smn2 gene.

Israel - English - Ministry of Health

novartis israel ltd - onasemnogene abeparvovec - suspension - onasemnogene abeparvovec 2 x 10^13 vg / 1 ml - onasemnogene abeparvovec - zolgensma is indicated for the treatment of:- patients with 5q spinal muscular atrophy (sma) with a bi-allelic mutation in the smn1 gene and a clinical diagnosis of sma type 1, or- patients with 5q sma with a bi-allelic mutation in the smn1 gene and up to 3 copies of the smn2 gene.

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

novartis pharmaceuticals australia pty ltd - onasemnogene abeparvovec, quantity: 1 u - injection, intravenous infusion - excipient ingredients: magnesium chloride hexahydrate; water for injections; sodium chloride; poloxamer; hydrochloric acid; trometamol - zolgensma (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (smn1) gene and 1 to 3 copies of the smn2 gene.

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

novartis pharmaceuticals australia pty ltd - onasemnogene abeparvovec, quantity: 1 u - injection, intravenous infusion - excipient ingredients: sodium chloride; water for injections; hydrochloric acid; trometamol; magnesium chloride hexahydrate; poloxamer - zolgensma (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (smn1) gene and 1 to 3 copies of the smn2 gene.

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

novartis pharmaceuticals australia pty ltd - onasemnogene abeparvovec, quantity: 1 u - injection, intravenous infusion - excipient ingredients: sodium chloride; poloxamer; magnesium chloride hexahydrate; water for injections; hydrochloric acid; trometamol - zolgensma (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (smn1) gene and 1 to 3 copies of the smn2 gene.

Canada - English - Health Canada

novartis pharmaceuticals canada inc - onasemnogene abeparvovec - solution - 20000000000000vg - onasemnogene abeparvovec 20000000000000vg - cellular and gene therapy

New Zealand - English - Medsafe (Medicines Safety Authority)

novartis new zealand ltd - onasemnogene abeparvovec 20tvg/ml (tvg = tera vector genomes; equivalent to 2 x 10^13 vector genomes per ml); onasemnogene abeparvovec 20tvg/ml (tvg = tera vector genomes; equivalent to 2 x 10^13 vector genomes per ml) - solution for infusion - active: onasemnogene abeparvovec 20tvg/ml (tvg = tera vector genomes; equivalent to 2 x 10^13 vector genomes per ml) excipient: hydrochloric acid magnesium chloride poloxamer 188 sodium chloride trometamol water for injection active: onasemnogene abeparvovec 20tvg/ml (tvg = tera vector genomes; equivalent to 2 x 10^13 vector genomes per ml) excipient: hydrochloric acid magnesium chloride poloxamer 188 sodium chloride trometamol water for injection - zolgensma is an adeno-associated virus (aav) vector-based gene therapy indicated for the treatment of paediatric patients with spinal muscular atrophy (sma) with a bi allelic mutation in the survival motor neuron 1 (smn1) gene.

New Zealand - English - Medsafe (Medicines Safety Authority)

novartis new zealand ltd - onasemnogene abeparvovec 20tvg/ml (tvg = tera vector genomes; equivalent to 2 x 10^13 vector genomes per ml) - solution for infusion - active: onasemnogene abeparvovec 20tvg/ml (tvg = tera vector genomes; equivalent to 2 x 10^13 vector genomes per ml) excipient: hydrochloric acid magnesium chloride poloxamer 188 sodium chloride trometamol water for injection - zolgensma is an adeno-associated virus (aav) vector-based gene therapy indicated for the treatment of paediatric patients with spinal muscular atrophy (sma) with a bi allelic mutation in the survival motor neuron 1 (smn1) gene.

United Arab Emirates - English - MOHAP (Ministry of Health & Prevention) - وزارة الصحة ووقاية المجتمع.الإمارات

1 kit (5.5 ml or 8.3 ml single use vial x 2 to 14) -