European Union - English - EMA (European Medicines Agency)

amicus therapeutics europe limited - cipaglucosidase alfa - glycogen storage disease type ii - other alimentary tract and metabolism products, - pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset pompe disease (acid α-glucosidase [gaa] deficiency).

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

amicus therapeutics us, llc - cipaglucosidase alfa (unii: 4sed7f4bsg) (cipaglucosidase alfa - unii:4sed7f4bsg) - pombiliti is indicated, in combination with opfolda, for the treatment of adult patients with late-onset pompe disease (lysosomal acid alpha-glucosidase [gaa] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ert). pombiliti in combination with opfolda is contraindicated in pregnancy [see warnings and precautions (5.4) and use in specific populations (8.1)]. risk summary based on findings from animal reproduction studies, pombiliti in combination with opfolda may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. in a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with cipaglucosidase alfa-atga in combination with miglustat at 16-fold and 3-fold, respectively, the mrhd of pombiliti and opfolda based on plasma auc exposure. a no observed adverse effect level (noael) was not identified for the combination. in a pre- and post-

European Union - English - EMA (European Medicines Agency)

amicus therapeutics europe limited - miglustat - glycogen storage disease type ii - other alimentary tract and metabolism products - opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset pompe disease (acid α- glucosidase [gaa] deficiency).

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

amicus therapeutics us, llc - miglustat (unii: adn3s497az) (miglustat - unii:adn3s497az) - opfolda is indicated, in combination with pombiliti, for the treatment of adult patients with late-onset pompe disease (lysosomal acid alpha-glucosidase [gaa] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ert). opfolda in combination with pombiliti is contraindicated in pregnancy [see use in specific populations (8.1)] risk summary based on findings from animal reproduction studies, opfolda in combination with pombiliti may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. in a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with miglustat in combination with cipaglucosidase alfa-atga at 3-fold and 16-fold, respectively, the mrhd of opfolda and pombiliti based on plasma auc exposure. a no observed adverse effect level (noael) was not identified for the combination. in a pre- and post-natal development study in rats, inc

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

sanofi-aventis australia pty ltd - avalglucosidase alfa, quantity: 100 mg - injection, powder for - excipient ingredients: histidine; polysorbate 80; mannitol; histidine hydrochloride monohydrate; glycine - nexviazyme is indicated for long-term enzyme replacement therapy for the treatment of patients one year of age and older with pompe disease (acid ?-glucosidase deficiency).

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

genzyme corporation - avalglucosidase alfa (unii: eo144cp0x9) (avalglucosidase alfa - unii:eo144cp0x9) - nexviazyme is indicated for the treatment of patients 1 year of age and older with late-onset pompe disease (lysosomal acid alpha-glucosidase [gaa] deficiency). none. risk summary available data from case reports of nexviazyme use in pregnant women are insufficient to evaluate for a drug associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. however, available data from postmarketing reports and published case reports on alglucosidase alfa (another hydrolytic lysosomal glycogen-specific enzyme replacement therapy) use in pregnant women have not identified a drug-associated risk of adverse pregnancy outcomes. the continuation of treatment for pompe disease during pregnancy should be individualized to the pregnant woman. untreated pompe disease may result in worsening disease symptoms in pregnant women (see clinical considerations) . embryo-fetal toxicity studies performed in pregnant mice resulted in maternal toxicity related to an immunologic response (including an anaphyl

European Union - English - EMA (European Medicines Agency)

sanofi b.v. - avalglucosidase alfa - glycogen storage disease type ii - other alimentary tract and metabolism products, - nexviadyme (avalglucosidase alfa) is indicated for long-term enzyme replacement therapy for the treatment of patients with pompe disease (acid α-glucosidase deficiency).

Canada - English - Health Canada

sanofi-aventis canada inc - avalglucosidase alfa - powder for solution - 100mg - avalglucosidase alfa 100mg - enzymes

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

genzyme corporation - alglucosidase alfa (unii: dti67o9503) (alglucosidase alfa - unii:dti67o9503) - alglucosidase alfa 5 mg in 1 ml - lumizyme® (alglucosidase alfa) [see description (11)] is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with pompe disease (acid α-glucosidase [gaa] deficiency). none. risk summary data from postmarketing reports and published case reports with alglucosidase alfa use in pregnant women have not identified a lumizyme-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. the continuation of treatment for pompe disease during pregnancy should be individualized to the pregnant woman. untreated pompe disease may result in worsening disease symptoms in pregnant women [see clinical considerations] . reproduction studies performed in mice and rabbits at doses resulting in exposures up to 0.4 or 0.5 times the human steady-state auc (area under the plasma concentration-time curve), respectively, during the period of organogenesis revealed no evidence of effects on embryo-fetal development. in mice there was an increase in pup mortality during lactation

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

sanofi-aventis australia pty ltd - alglucosidase alfa, quantity: 52.5 mg - injection, powder for - excipient ingredients: monobasic sodium phosphate monohydrate; polysorbate 80; mannitol; nitrogen - myozyme (alglucosidase alfa-rch) is indicated for the long-term treatment of patients with a confirmed diagnosis of pompe disease (acid alfa-glucosidase deficiency).