Združene države Amerike - angleščina - NLM (National Library of Medicine)

alexion pharmaceuticals inc. - ravulizumab (unii: c3vx249t6l) (ravulizumab - unii:c3vx249t6l) - ultomiris is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (pnh). ultomiris is indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (ahus) to inhibit complement-mediated thrombotic microangiopathy (tma). limitations of use: ultomiris is not indicated for the treatment of patients with shiga toxin e. coli related hemolytic uremic syndrome (stec-hus). ultomiris is indicated for the treatment of adult patients with generalized myasthenia gravis (gmg) who are anti-acetylcholine receptor (achr) antibody-positive. ultomiris is indicated for the treatment of adult patients with neuromyelitis optica spectrum disorder (nmosd) who are anti-aquaporin-4 (aqp4) antibody positive. ultomiris is contraindicated for initiation in patients with unresolved serious neisseria meningitidis infection [see warnings and precautions (5.1)] . risk summary there are no available data on ultomiris use in pregnant women to inform 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 and ahus in pregnancy (see clinical considerations ). animal studies using a mouse analogue of the ravulizumab-cwvz molecule (murine anti-mouse complement component 5 [c5] antibody) showed increased rates of developmental abnormalities and an increased rate of dead and moribund offspring at doses 0.8-2.2 times the human dose (see data). the estimated background risk of major birth defects and miscarriage for the indicated populations 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. in pregnancy, ahus is associated with adverse maternal outcomes, including preeclampsia and preterm delivery, and adverse fetal/neonatal outcomes, including intrauterine growth restriction (iugr), fetal death and low birth weight. data animal data animal reproduction studies were conducted in mice using doses of a murine anti-c5 antibody that approximated 1-2.2 times (loading dose) and 0.8-1.8 times (maintenance dose) the recommended human ultomiris dose, based on a body weight comparison. when animal exposure to the antibody occurred in the time period from before mating until early gestation, no decrease in fertility or reproductive performance was observed. when maternal exposure to the antibody occurred during organogenesis, 2 cases of retinal dysplasia and 1 case of umbilical hernia were observed among 230 offspring born to mothers exposed to the higher antibody dose; however, the exposure did not increase fetal loss or neonatal death. when maternal exposure to the antibody occurred in the time period from implantation through weaning, a higher number of male offspring became moribund or died (1/25 controls, 2/25 low dose group, 5/25 high dose group). surviving offspring had normal development and reproductive function. human igg are known to cross the human placental barrier, and thus ultomiris may potentially cause terminal complement inhibition in fetal circulation. risk summary there are no data on the presence of ravulizumab-cwvz in human milk, the effect on the breastfed child, or the effect on milk production. since many medicinal products and immunoglobulins are secreted into human milk, and because of the potential for serious adverse reactions in a nursing child, breastfeeding should be discontinued during treatment and for 8 months after the final dose. the safety and effectiveness of ultomiris administered intravenously for the treatment of pnh have been established in pediatric patients aged one month and older. use of ultomiris for this indication is supported by evidence from adequate and well-controlled trials in adults with additional pharmacokinetic, efficacy and safety data in pediatric patients aged 9 to 17 years [see adverse reactions (6.1), clinical pharmacology (12.3), and clinical studies (14.1)] . the safety and efficacy for the treatment of pediatric and adult patients with pnh appear similar. use of ultomiris administered intravenously in pediatric patients with pnh aged less than 9 years and body weight < 30 kg is based on extrapolation of pharmacokinetic / pharmacodynamic (pk/pd), and efficacy and safety data from ahus and pnh clinical studies [see clinical pharmacology (12.3) and clinical studies (14)] . the safety and effectiveness of ultomiris administered intravenously for the treatment of ahus have been established in pediatric patients aged one month and older. use of ultomiris for this indication is supported by evidence from adequate and well-controlled studies in adults with additional pharmacokinetic, safety, and efficacy data in pediatric patients aged 10 months to < 17 years. the safety and efficacy of ultomiris administered intravenously for the treatment of ahus appear similar in pediatric and adult patients [see adverse reactions (6.1), and clinical studies (14.2)] . the safety and effectiveness of ultomiris for the treatment of gmg or nmosd in pediatric patients have not been established. subcutaneous administration of ultomiris has not been evaluated and is not approved for use in pediatric patients. clinical studies of ultomiris did not include sufficient numbers of subjects aged 65 and over (58 patients with pnh, 9 with ahus, 54 with gmg, and 7 with nmosd) to determine whether they respond differently from younger subjects. other reported clinical experience has not identified differences in responses between elderly and younger patients. - it is important that you receive training from your healthcare provider on how to inject ultomiris before giving an injection. - if you have a serious allergic reaction (such as chest pain, trouble breathing, facial swelling, and/or feeling faint), remove the on-body injector(s) to stop the injection and get medical help right away. - you will need 2 ultomiris on-body injectors and 2 prefilled cartridges to receive your full dose. - store ultomiris injector and cartridge in the refrigerator between 36°f to 46°f (2°c to 8°c). - keep the injector and cartridge in the original carton to protect from light or physical damage. - for your injection, take 2 injectors and 2 cartridges out of the refrigerator and let them sit at room temperature for at least 45 minutes before you inject. - do not return to the refrigerator. the injector and cartridge may be stored in the original carton box at room temperature between 68°f to 77°f (20°c to 25°c) for up to 3 days. discard (throw away) after 3 days if unused. - do not freeze. - keep ultomiris and all medicines out of the reach of children. - do not shake or drop the injector or cartridge. - do not remove the injector or cartridge from the carton or clear tray until you are ready to inject. - do not press the blue start button on the injector until you place the loaded injector onto your skin and are ready to inject. - after you insert the cartridge into the injector, make sure you inject within 5 minutes. loading the cartridge more than 5 minutes before your injection can dry out the medicine. - do not use the injector or cartridge if the packaging appears to be opened, or if the injector or cartridge has been dropped or appears to be broken or damaged. part of the on-body injector or prefilled cartridge may be broken even if you cannot see the damage. - do not reuse the on-body injector and prefilled cartridge. they are single-use only. - do not let the injector get wet from water or other liquids. it contains electronic parts that should not get wet. - keep the on-body injector a minimum of 4 inches (10 cm) away from other electronics such as cellular phones. - do not use the injector or cartridge past the expiration date printed on the carton and cartridge. - do not use a microwave, hot water, hair dryer, or any other heat sources to warm the prefilled cartridge. - do not return to the refrigerator. the injectors and cartridges may be stored in the original carton box at room temperature between 68°f to 77°f (20°c to 25°c) for up to 3 days. throw away (discard) after 3 days if unused. - do not touch the blue start button until the injectors are on your skin and you are ready to inject. - do not use if the white paper cover(s) or plastic cover(s) is missing or damaged. if either is missing, call 1-888-765-4747. - 2 clear trays containing the on-body injectors and prefilled cartridges (see figure e) - alcohol wipes - cotton ball or gauze pad - adhesive bandage - sharps disposal container - do not inject into areas of the skin that are tender, bruised, red or hard, or areas with wrinkles, skin folds, scars, tattoos, stretch marks, moles, or excessive hair. - do not use the same sites 2 weeks in a row. change (rotate) your injection sites every week to reduce irritation. 2a - do not use if the medicine is cloudy, discolored, or contains flakes or particles. - do not use if any part of cartridge looks cracked, broken, or if pieces are missing. - do not hold it by the ends. - do not turn (rotate) or remove the top or bottom of the cartridge. - do not touch the cartridge bottom after cleaning. - note: it is okay to see a few drops of the medicine coming out of the needle. - do not pull the skin adhesive backing off of the injector. - do not touch the skin adhesive. - do not touch the start button until you have placed the loaded injector onto your skin. - to prevent injury, do not touch the needle cover area. - do not place the loaded injector on your body if the red status light flashes continuously. - do not fold the skin adhesive over onto itself. - you can see the status light. - the injectors are at least 1 inch (2.5 cm) apart. - clothing is kept away from adhesive. - do not move the loaded injector after it has been placed onto your skin. - you will hear 3 "beeps" when the blue button is pressed and injection starts. - the status light will flash green during the injection. - the sound of the injector motor will be heard for about 10 minutes during the injection. - you may feel a pinch. - light physical activities can be done during injection, such as walking, reaching, and bending. - the injector should stay dry. do not let the injector get wet from water or other liquids. it contains electronic parts that should not get wet. - status light turns solid green. - begins "beeping" 3 times every 30 seconds. - white plunger fills the medicine window. (see figure q) - put the used ultomiris on-body injectors and prefilled cartridges in an fda-cleared sharps disposal container right away after use. do not throw away (dispose of) the ultomiris on-body injectors and prefilled cartridges in your household trash. - if you do not have an fda-cleared sharps disposal container, you may use a household container that is: - made of a heavy-duty plastic, - can be closed with a tight-fitting, puncture-resistant lid, without sharps being able to come out, - upright and stable during use, - leak-resistant, and - properly labeled to warn of hazardous waste inside the container. - when your sharps disposal container is almost full, you will need to follow your community guidelines for the right way to dispose of your sharps disposal container. there may be state or local laws about how you should throw away needles and syringes. for more information about safe sharps disposal, and for specific information about sharps disposal in the state you live in, go to the fda's website at: http://www.fda.gov/safesharpsdisposal - do not recycle your used sharps disposal container. - if there is blood at the injection sites, press a cotton ball or gauze pad on site. - apply an adhesive bandage if necessary. - do not rub the injection site. frequently asked questions - you need 2 on-body injectors and 2 prefilled cartridges for a full weekly dose. you may prepare and use 2 on-body injectors and 2 prefilled cartridges at the same time. - if the status light flashes red at any time, this means there is an error and the on-body injector will no longer work. remove the on-body injector from the body (if it is attached) and place it in the original packaging. do not remove the prefilled cartridge from the on-body injector. contact 1-888-765-4747 and return the on-body injector and cartridge per the instructions given. - see step 2a and figure i. if you still cannot open it, contact 1-888-765-4747. - first check that both pull tabs on the back of the on-body injector have been fully removed, including the battery strip. if the on-body injector still does not turn on, use a new on-body injector and prefilled cartridge. contact 1-888-765-4747. - if you removed the pull tabs and pressed the start button, the on-body injector will make a beeping sound and you will see the status light flash red. the on-body injector will no longer work. stop using the on-body injector and contact 1-888-765-4747. - make sure you firmly press the blue start button. if it still does not start, remove the on-body injector and contact 1-888-765-4747. do not reapply the same on-body injector as it will not work. use a new on-body injector and prefilled cartridge. - do not use the on-body injector or prefilled cartridge and contact 1-888-765-4747. - it is important to inject within 5 minutes after loading the prefilled cartridge. - after you load the prefilled cartridge into the on-body injector, it is completely normal to see the medicine drop out of the needle. this is to get rid of the air from the on-body injector needle. however, if you wait more than 5 minutes to start your injection, you may lose some of your medicine. additionally, this may dry out the medicine and clog the on-body injector needle. - do not return to the refrigerator the on-body injectors and prefilled cartridges after they reach room temperature. this may degrade the medicine. if needed, the on-body injectors and prefilled cartridges may be stored in the original carton box at room temperature between 68°f to 77°f (20°c to 25°c) for up to 3 days. throw away after 3 days if unused. - relative humidity range is 15% to 85%. - altitude range is -984 feet to 11483 feet (-300 meters to 3500 meters). - during injection, keep the on-body injector a minimum of 4 inches (10 cm) away from other electronics such as cellular phones. - warning: do not modify the on-body injector or prefilled cartridge. - warning: magnetic resonance (mr) is unsafe, the on-body injector for ultomiris should not enter the mr scanning room. - on-body injector operating temperature range is 59°f to 104°f (15°c to 40°c).

Združene države Amerike - angleščina - NLM (National Library of Medicine)

novartis pharmaceuticals corporation - iptacopan hydrochloride (unii: xw5ck7c6yh) (iptacopan - unii:8e05t07z6w) - fabhalta is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (pnh). fabhalta is contraindicated: - in patients with serious hypersensitivity to iptacopan or any of the excipients. - for initiation in patients with unresolved serious infection caused by encapsulated bacteria, including streptococcus pneumoniae, neisseria meningitidis, or haemophilus influenzae type b. risk summary available data from clinical trials with fabhalta use in pregnant women are insufficient to identify a drug-associated risk of major birth defects, miscarriage, or other 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 fabhalta 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 iptacopan to pregnant rats and rabbits during organogenesis at exposures 4 to 6-times the human exposure (based on auc) at the maximum recommended human dose (mrhd) of 200 mg twice daily did not induce embryo or fetal toxicity (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 major birth defects, loss, or other adverse outcomes. in the u.s. general population, the estimated background risk of major birth defects and miscarriages in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. clinical considerations disease-associated maternal and/or embryo/fetal risk pnh in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombosis, infections, bleeding, miscarriages, increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery. data animal data in an embryo-fetal development study in rats, oral administration of iptacopan during organogenesis did not cause embryo-fetal toxicity when given up to the highest dose of 1,000 mg/kg/day, which corresponds to 4-times the mrhd based on auc. in an embryo-fetal development study in rabbits, oral administration of iptacopan during organogenesis did not cause embryo-fetal toxicity when given up to the highest dose of 450 mg/kg/day, which corresponds to 6-times the mrhd based on auc. in a pre- and postnatal development study in rats, oral administration of iptacopan during gestation, parturition, and lactation did not cause adverse effects in offspring when given up to the highest dose of 1,000 mg/kg/day, which corresponds to 4-times the mrhd based on auc. risk summary there are no data on the presence of iptacopan or its metabolite in either human or animal milk, the effects on the breastfed child or on milk production. since many medicinal products are secreted into human milk, and because of the potential for serious adverse reactions in a breastfed child, breastfeeding should be discontinued during treatment and for 5 days after the final dose. safety and effectiveness in pediatric patients with pnh have not been established. there were 29 pnh patients 65 years of age and older in apply-pnh and appoint-pnh [see clinical studies (14)] . of the total number of fabhalta-treated patients during the 24-week treatment period in these studies, 21 (20.6%) were 65 years of age and older, while 7 (6.9%) were 75 years of age and older. clinical studies of fabhalta did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. the use of fabhalta is not recommended in patients with severe renal impairment (estimated glomerular filtration rate (egfr) < 30 ml/min/1.73 m2 ) with or without hemodialysis. no dose adjustment is required in patients with mild (egfr 60 to < 90 ml/min/1.73 m2 ) or moderate (egfr 30 to < 60 ml/min/1.73 m2 ) renal impairment [see clinical pharmacology (12.3)] . the use of fabhalta is not recommended in patients with severe hepatic impairment (child-pugh class c). no dose adjustment is required for patients with mild (child-pugh class a) or moderate (child-pugh class b) hepatic impairment [see clinical pharmacology (12.3)] .

Avstralija - angleščina - Department of Health (Therapeutic Goods Administration)

glaxosmithkline australia pty ltd - haemophilus influenza type b polyribose ribitol phosphate,meningococcal polysaccharide group c,tetanus toxoid -

Izrael - angleščina - Ministry of Health

sanofi israel ltd - meningococcal vaccines group a; meningococcal vaccines group c; meningococcal vaccines group w; meningococcal vaccines group y - solution for injection - meningococcal vaccines group c 4 mcg / 0.5 ml; meningococcal vaccines group y 4 mcg / 0.5 ml; meningococcal vaccines group w 4 mcg / 0.5 ml; meningococcal vaccines group a 4 mcg / 0.5 ml - other meningococcal monovalent purified polysaccharides antigen - other meningococcal monovalent purified polysaccharides antigen - active immunization of individuals 9 months through 55 years of age for the prevention of invasive meningococcal disease caused by n meningitidis serogroups a, c, y, and w-135 menactra vaccine does not prevent n meningitidis serogroup b disease.

Irska - angleščina - HPRA (Health Products Regulatory Authority)

glaxosmithkline (ireland) limited - conjugate of haemophilus influenzae type b capsular polysaccharide (polyribosylribitol phosphate) and tetanus; conjugate of neisseria meningitides c capsular polysaccharide and tetanus toxoid (mean tt/ps ratio :1) - powder and solvent for solution for injection - 0.5 millilitre(s) - hemophilus influenzae b, combinations with meningococcus c, conjugated

Evropska unija - angleščina - EMA (European Medicines Agency)

gsk vaccines s.r.l. - outer membrane vesicles from neisseria meningitidis group b (strain nz 98/254), recombinant neisseria meningitidis group b fhbp fusion protein, recombinant neisseria meningitidis group b nada protein, recombinant neisseria meningitidis group b nhba fusion protein - meningitis, meningococcal - meningococcal vaccines - active immunisation against invasive disease caused by neisseria meningitidis serogroup-b strains.,

Evropska unija - angleščina - EMA (European Medicines Agency)

pfizer europe ma eeig - neisseria meningitidis serogroup b fhbp (recombinant lipidated fhbp (factor h binding protein)) subfamily a; neisseria meningitidis serogroup b fhbp (recombinant lipidated fhbp (factor h binding protein)) subfamily b - meningitis, meningococcal - bacterial vaccines, meningococcal vaccines - trumenba is indicated for active immunisation of individuals 10 years and older to prevent invasive meningococcal disease caused by neisseria meningitidis serogroup b.the use of this vaccine should be in accordance with official recommendations.

Avstralija - angleščina - Department of Health (Therapeutic Goods Administration)

glaxosmithkline australia pty ltd - meningococcal oligosaccharide group y, quantity: 5 microgram; meningococcal oligosaccharide group c, quantity: 5 microgram; diphtheria crm197 protein, quantity: 16 microgram; meningococcal oligosaccharide group w135, quantity: 5 microgram - injection, solution - excipient ingredients: sodium chloride; water for injections; monobasic sodium phosphate monohydrate; dibasic sodium phosphate dihydrate - menveo is indicated for active immunisation of infants and children (from 2 months of age), adolescents and adults to prevent invasive disease caused by neisseria meningitidis serogroups a, c, w-135 and y. the use of this vaccine should be in accordance with official recommendations.

Avstralija - angleščina - Department of Health (Therapeutic Goods Administration)

glaxosmithkline australia pty ltd - neisseria meningitidis group b neisseria heparin binding antigen fusion protein, quantity: 50 microgram; neisseria meningitidis serogroup b outer membrane vesicles, quantity: 25 microgram; neisseria meningitidis group b factor h binding protein fusion protein, quantity: 50 microgram; neisseria meningitidis group b neisseria adhesin a protein, quantity: 50 microgram - injection, suspension - excipient ingredients: water for injections; aluminium hydroxide hydrate; histidine; sodium chloride; sucrose - bexsero is indicated for active immunisation against invasive disease caused by n. meningitidis group b strains. for information on protection against specific group b strains see section 5.1 pharmacodynamic properties. bexsero is indicated for vaccination of individuals from 2 months of age and older.

Avstralija - angleščina - Department of Health (Therapeutic Goods Administration)

glaxosmithkline australia pty ltd - neisseria meningitidis group b factor h binding protein fusion protein, quantity: 50 microgram; neisseria meningitidis group b neisseria adhesin a protein, quantity: 50 microgram; neisseria meningitidis serogroup b outer membrane vesicles, quantity: 25 microgram; neisseria meningitidis group b neisseria heparin binding antigen fusion protein, quantity: 50 microgram - injection, suspension - excipient ingredients: sucrose; histidine; water for injections; aluminium hydroxide hydrate; sodium chloride - bexsero is indicated for active immunisation against invasive disease caused by n. meningitidis group b strains. for information on protection against specific group b strains see section 5.1 pharmacodynamic properties. bexsero is indicated for vaccination of individuals from 2 months of age and older.