Bandaríkin - enska - NLM (National Library of Medicine)

alexion pharmaceuticals inc. - danicopan (unii: jm8c1sfx0u) (danicopan - unii:jm8c1sfx0u) - voydeya is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (evh) in adults with paroxysmal nocturnal hemoglobinuria (pnh). limitations of use voydeya has not been shown to be effective as monotherapy and should only be prescribed as an add-on to ravulizumab or eculizumab. voydeya is contraindicated for initiation in patients with unresolved serious infection caused by encapsulated bacteria, including neisseria meningitidis , streptococcus pneumoniae , or haemophilus influenzae type b [see warnings and precautions (5.1)] . risk summary there are no available data on voydeya use in pregnant individuals to evaluate for a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes. there are risks to the mother and fetus associated with untreated pnh in pregnancy (see clinical considerations) . the use of voydeya in pregnant women or women planning to become pregnant may be considered following an assessment of the risks and benefits. in animal reproduction studies, oral administration of danicopan to pregnant new zealand white (nzw) rabbits and wistar hans (wh) rats during organogenesis at exposures 18 or 25-times, respectively, above the human exposure at the maximum recommended human dose (mrhd) of 200 mg three times a day (based on auc) resulted in no adverse developmental effects (see data) . the estimated background risk of major birth defects and miscarriage for the indicated population is unknown. all pregnancies have a background risk of birth defect, loss, or other adverse outcomes. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. clinical considerations disease-associated maternal and/or fetal/neonatal risk pnh in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombotic events, infections, bleeding, miscarriages, and increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery. data animal data there were no effects on early embryonic development and fetal development in nzw rabbits (where danicopan is pharmacodynamically active) up to a mean maternal systemic exposure 18-times the exposure at the mrhd (based on auc) or during post-natal development up to a mean maternal systemic exposure 9-times the exposure at the mrhd (based on auc). in wh rats (where danicopan lacks pharmacodynamic activity), there were no effects on embryo-fetal development up to a mean maternal exposure 25-times the exposure at the mrhd (based on auc). risk summary there are no data on the presence of danicopan in human milk, the effects on the breastfed child, or the effect on milk production. danicopan is present in animal milk. when a drug is present in animal milk, it is likely that the drug will be present in human milk. because of the potential for serious adverse reactions in the breastfed child, including serious infections with encapsulated bacteria and liver enzyme increases, advise patients not to breastfeed during treatment with voydeya, and for 3-days after the last dose. data animal data danicopan was excreted into the milk of lactating rabbits following oral administration from lactation day 4 to lactation day 10, with mean milk concentrations at approximately 2 hours following dose administration 5- and 3.5-times higher than the mean maternal plasma concentrations at 50 and 250 mg/kg/day, respectively. mean milk concentrations in dams were 19- and 43-times higher than the systemic exposure at the mrhd (based on rabbit concentration at 2 hours vs. human cmax ). safety and effectiveness of voydeya for the treatment of pnh in pediatric patients have not been established. there were 22 patients 65 years of age and older in the clinical studies for pnh [see clinical studies (14)] . of the total number of voydeya-treated patients in these studies, 16 (28.1%) were 65 years of age and older, and 7 (12.3%) were 75 years of age and older. clinical studies of voydeya did not include sufficient numbers of subjects aged 65 years and over to determine whether they respond differently from younger subjects. no dose adjustment is required in patients with mild to moderate hepatic impairment (child-pugh class a and b). studies have not been conducted in patients with severe hepatic impairment, therefore, avoid use of voydeya in this patient population [see warnings and precautions (5.3)] .

Ástralía - enska - Department of Health (Therapeutic Goods Administration)

swedish orphan biovitrum pty ltd - pegcetacoplan, quantity: 1080 mg - injection, solution - excipient ingredients: water for injections; sodium hydroxide; sodium acetate trihydrate; glacial acetic acid; sorbitol - empaveli is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (pnh) who have an inadequate response to, or are intolerant of, a c5 inhibitor.

Kanada - enska - Health Canada

swedish orphan biovitrum ab (publ) - pegcetacoplan - solution - 1080mg - pegcetacoplan 1080mg