miglustat

HOS-288451 · has-adverse-reaction-information · PDF-Seite 5

Summary of the safety profile The most commonly reported adverse reaction only attributable to miglustat 65 mg was constipation (1.3%). Tabulated list of adverse reactions The assessment of adverse reactions was informed by subjects treated with miglustat in combination with cipaglucosidase alfa therapy from the pooled safety analysis across the 3 clinical trials. The total mean duration of exposure was 28.0 months. Adverse reactions are listed by MedDRA system organ class in Table 1. The corresponding frequency categories are defined as follows: very common (≥ 1/10), common (≥ 1/100 to < 1/10), uncommon (≥ 1/1 000 to < 1/100), rare (≥ 1/10 000 to < 1/1 000), very rare (< 1/10 000), and not known (cannot be estimated from available data). Table 1: Summary of adverse reactions of miglustat-treated patients System organ class (SOC) Frequency Adverse reaction (preferred term) Immune system disorders Common Anaphylactic reaction7 Uncommon Hypersensitivity Nervous system disorders Very common Headache Common Tremor, dysgeusia, paraesthesia Uncommon Balance disorder, migraine4 Cardiac disorders Common Tachycardia6 Vascular disorders Common Hypotension Uncommon Pallor Respiratory, thoracic and Common Dyspnoea mediastinal disorders Uncommon Asthma Gastrointestinal disorders Common Diarrhoea, nausea, abdominal pain1, flatulence, abdominal distension, vomiting, constipation† Uncommon Abdominal discomfort†, oesophageal spasm, oral pain Skin and subcutaneous tissue Common Urticaria3, rash2, pruritus, hyperhidrosis disorder Uncommon Skin discolouration Musculoskeletal and connective Common Muscle spasms, myalgia, arthralgia, muscular tissue disorders weakness 5  System organ class (SOC) Frequency Adverse reaction (preferred term)

Auszug; vollständigen Abschnitt im Original lesen.

HOS-288447 · has-contraindication-information · PDF-Seite 4

Hypersensitivity to the active substance or to any of the excipients listed in section 6.1. Contraindication to cipaglucosidase alfa.

HOS-288446 · has-indication-information · PDF-Seite 2

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).

HOS-288449 · has-interaction-information · PDF-Seite 4

No interaction studies have been performed related to the use of miglustat. Food interaction Miglustat is known to have a direct effect on the enzymatic function of major disaccharidases of the intestinal epithelium. Specifically, miglustat inhibits disaccharidases with alpha-glycosidic linkages including sucrase, maltase, and isomaltase. The strength of potential interactions can immediately interfere with digestive activity of sucrose, maltose and isomaltose leading to maldigestion, osmotic influx of water, increased fermentation, and production of irritating metabolites. Patients should fast for 2 hours before and for 2 hours after taking miglustat.

HOS-288450 · has-pregnancy-information · PDF-Seite 4

Contraception in females Reliable contraceptive measures must be used by women of childbearing potential during treatment with miglustat in combination with cipaglucosidase alfa, and for 4 weeks after discontinuing treatment (see section 5.3). The medicinal product is not recommended in women of childbearing potential not using reliable contraception. Pregnancy There are no clinical data from the use of miglustat in combination with cipaglucosidase alfa in pregnant women. Miglustat crosses the placenta. Animal studies with miglustat in combination with cipaglucosidase alfa as well as with miglustat alone have shown reproductive toxicity (see section 5.3). Miglustat in combination with cipaglucosidase alfa therapy is not recommended during pregnancy. Breast-feeding It is not known if miglustat and cipaglucosidase alfa are secreted in human breast milk (see section 5.3). Available pharmacodynamic/toxicological data in animals have shown secretion of miglustat and excretion of cipaglucosidase alfa in milk. A risk to newborns/infants cannot be excluded. A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from miglustat in combination with cipaglucosidase alfa therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman. Fertility There are no clinical data on the effects of miglustat in combination with cipaglucosidase alfa therapy on fertility. 4 No effects on sperm concentration, motility, or morphology were seen in 7 healthy adult men who received miglustat 100 mg, orally, twice daily for 6 weeks. In male rats, no effect on spermatogenesis was observed following administration of miglustat in combination with cipaglucosidase alfa or miglustat alone. However, preclinical data from a study in rats using another miglustat product have shown that miglustat adversely affects sperm parameters (motility and morphology), thereby reducing fertility (see section 5.3). In female rats, increase in pre-implantation loss was noted with miglustat in combination with cipaglucosidase alfa and with miglustat alone (see section 5.3).

HOS-288448 · has-warning-information · PDF-Seite 4

Adverse drug reactions may occur upon the use of miglustat in combination with cipaglucosidase alfa (see section 4.8).