الاتحاد الأوروبي - الإنجليزية - EMA (European Medicines Agency)

samsung bioepis nl b.v. - eculizumab - hemoglobinuria, paroxysmal - immunosuppressants - epysqli is indicated in adults and children for the treatment of paroxysmal nocturnal haemoglobinuria (pnh).evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history.

أستراليا - الإنجليزية - Department of Health (Therapeutic Goods Administration)

alexion pharmaceuticals australasia pty ltd - eculizumab -

الولايات المتحدة - الإنجليزية - NLM (National Library of Medicine)

apellis pharmaceuticals, inc. - pegcetacoplan (unii: to3jyr3bou) (pegcetacoplan - unii:to3jyr3bou) - empaveli® is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (pnh). empaveli is contraindicated: - in patients with hypersensitivity to pegcetacoplan or to any of the excipients [see warnings and precautions (5.3)] . - for initiation in patients with unresolved serious infection caused by encapsulated bacteria including streptococcus pneumoniae , neisseria meningitidis , and haemophilus influenzae type b [see warnings and precautions (5.1)] . risk summary there are insufficient data on empaveli 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 in pregnancy (see clinical considerations) . the use of empaveli may be considered following an assessment of the risks and benefits. treatment of pregnant cynomolgus monkeys with pegcetacoplan at a subcutaneous dose of 28 mg/kg/day (2.9 times human exposure based on auc) from the gestation period through parturition resulted in a statistically significant increase in abortions or stillbirths compared to controls (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 defect, 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 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 animal reproduction studies with pegcetacoplan were conducted in cynomolgus monkeys. pegcetacoplan treatment of pregnant cynomolgus monkeys at a subcutaneous dose of 28 mg/kg/day (2.9 times human exposure based on auc) from the gestation period through parturition resulted in a statistically significant increase in abortions and stillbirths compared to controls. no increase in abortions or stillbirths occurred at a dose of 7 mg/kg/day (1.3 times human exposure based on auc). no maternal toxicity or teratogenic effects were observed in offspring delivered at term. no developmental effects were observed in infants up to 6 months postpartum. systemic exposure to pegcetacoplan of less than 1% of maternal levels was detected in fetuses from monkeys treated with 28 mg/kg/day from the period of organogenesis through the second trimester. risk summary it is not known whether pegcetacoplan is secreted in human milk or whether there is potential for absorption and harm to the infant. there are no data on the effects of pegcetacoplan on milk production. pegcetacoplan is present in milk of lactating monkeys (see animal data) . since many medicinal products are secreted into human milk, and because of the potential for serious adverse reaction in a breastfeeding child, breastfeeding should be discontinued during treatment and for 40 days after the last dose. data animal data pegcetacoplan was detectable in milk of lactating monkeys at less than 1% concentration of serum levels but was not detectable in the serum of nursing infants. contraception females empaveli may cause embryo-fetal harm when administered to pregnant women [see use in specific populations (8.1)] . pregnancy testing is recommended for females of reproductive potential prior to treatment with empaveli. advise female patients of reproductive potential to use effective contraception during treatment with empaveli and for 40 days after the last dose. safety and effectiveness in pediatric patients have not been established. clinical studies of empaveli did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. other reported clinical experience has not identified differences in responses between geriatric and younger patients. instructions for use empaveli® (em-puh-vel-ee) (pegcetacoplan) injection, for subcutaneous use infusion pump important information this instructions for use is for the infusion pump only. if using empaveli injector, follow the instructions for use that comes with the empaveli injector. read this instructions for use before you start using empaveli with an infusion pump and each time you get a refill as there may be new information. this information does not take the place of talking to your healthcare provider about your medical condition or treatment. your healthcare provider should show you or your caregiver how to infuse empaveli the right way before you use it for the first time. ask your healthcare provider about any instructions you do not understand. how should i store empaveli? - store vials of empaveli in the refrigerator between 36°f to 46°f (2°c to 8°c) in the original carton to protect from light. - do not use empaveli past the expiration date stamped on the carton. keep empaveli and all medicines out of the reach of children. - find a well-lit, flat work surface area, like a table. - remove a single vial carton from the refrigerator. keep the vial in the carton at room temperature and allow it to warm up for about 30 minutes. - do not try to speed up the warming process. - infusion pump and manufacturer's instructions (not shown) - compatible syringe for your infusion pump - transfer needle or - needleless transfer device to draw up the medicine from the vial - infusion set (not shown; varies according to device manufacturer's instructions) - infusion tubing - sharps container - alcohol wipes - gauze and tape, or transparent dressing - remove the vial from the carton. carefully look at the liquid in the vial of empaveli. empaveli is a clear, colorless to slightly yellowish liquid. check for particles or color changes (see figure b ). - empaveli is a clear, colorless to slightly yellowish liquid. check for particles or color changes (see figure b ). - the liquid looks cloudy, contains particles, or is dark yellow. - the protective flip cap is missing or damaged. - the expiration date on the label has passed. - remove the protective flip cap from the top of the vial to show the middle part of the gray rubber stopper of the empaveli vial (see figure c ). throw away the protective flip cap. - clean the gray rubber stopper with a new alcohol wipe and allow the gray rubber stopper to dry for at least 30 seconds. do not touch the exposed gray rubber stopper after wiping. - attach a sterile transfer needle to a sterile syringe. - pull back the plunger to the 20-ml mark to fill the syringe with air (see figure d ). - push the air-filled syringe with transfer needle attached down through the center of the vial gray rubber stopper. - the tip of the transfer needle should not be in the solution to avoid creating air bubble(s) (see figure e ). - gently push the air from the syringe into the vial. this will inject the air from the syringe into the vial. - turn the vial upside down and insert the transfer needle in the empaveli solution (see figure f ). - with the transfer needle tip in the empaveli solution, slowly pull the plunger back to fill the syringe with all the empaveli solution (see figure g ). - remove the filled syringe with empaveli and the transfer needle from the vial. - remove the transfer needle by using 1 hand to slide the needle into the needle cap and scoop upwards to cover the needle (see figure h ). - after the needle is covered, push the needle cap down towards the syringe to fully attach it with 1 hand to prevent an accidental stick with the needle (see figure i ). - twist off and remove the transfer needle (see figure j ). - gather the infusion pump supplies and follow the device manufacturer's instructions to prepare the pump and tubing. - select an area on your stomach (abdomen), thighs, hips, or upper arms for the infusion(s) (see figure k ). avoid the following infusion areas: do not infuse into areas where the skin is tender, bruised, red, or hard. avoid infusing into tattoos, scars, or stretch marks. - do not infuse into areas where the skin is tender, bruised, red, or hard. - avoid infusing into tattoos, scars, or stretch marks. - use a different site(s) from the last time you infused empaveli. if there are multiple infusion sites, they should be at least 3 inches apart. change (rotate) infusion sites in between each infusion (see figure l ). - clean the skin at each infusion site(s) with a new alcohol wipe, starting at the center of each infusion site and working outward in a circular motion (see figure m ). - let the skin dry. - pinch the skin between your thumb and forefinger around the infusion site (where you plan to insert the needle). - insert the needle into the skin (see figure n ). - secure the needle(s) using gauze and tape or a transparent dressing placed over the infusion site(s) (see figure o ). - follow the device manufacturer's instructions to start the infusion. - start the infusion right away after drawing empaveli into the syringe. - empaveli infusion takes about 30 minutes (if using 2 infusion sites) or about 60 minutes (if using 1 infusion site) to complete. - follow the device manufacturer's instructions to complete the infusion. - record your treatment as directed by your healthcare provider. - after the infusion is complete, remove the dressing and slowly take out the needle(s). cover the infusion site with a new dressing. - remove the infusion set from the pump and throw it away into the sharps container (see figure p ). - clean and store the infusion pump according to the device manufacturer's instructions. - put the used needles, syringes, and empaveli infusion tubing in an fda-cleared sharps disposal container right away after use (see figure p ). - do not dispose of (throw away) the used needles, syringes, and empaveli infusion tubing 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 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. - made of 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 used needles and syringes. - for more information about safe sharps disposal, and for specific information about sharps disposal in the state that you live in, go to the fda's website at: http://www.fda.gov/safesharpsdisposal. - do not throw away your used sharps disposal container in your household trash unless your community guidelines permit this. - do not recycle your used sharps disposal container. call 1-866-692-7527 to speak with an apellis representative. manufactured for: apellis pharmaceuticals, inc. 100 fifth avenue waltham, ma 02451 copyright © 2021 apellis pharmaceuticals, inc. all rights reserved. empaveli is a registered trademark of apellis pharmaceuticals, inc. this instructions for use has been approved by the u.s. food and drug administration. revised 09/2023 emp-ifu-29sep2023-4.0 instructions for use empaveli® injector (pegcetacoplan) injection, for subcutaneous use single-use on-body injector important information this instructions for use is for the empaveli injector only. read this instructions for use before you start using the injector and each time you get a refill as there may be new information. the empaveli injector is placed on your body to give medicine under the skin. this information does not take the place of talking to your healthcare provider about your medical condition or treatment. your healthcare provider should show you or your caregiver how to inject empaveli the right way before you use it for the first time. it is important that you do not try to give yourself or someone else the injection unless you have received training from your healthcare provider. ask your healthcare provider about any instructions you do not understand. if you have questions, concerns, or need of help, please call apellisassist® at 1-866-my-apl-assist (1-866-692-7527). how should i store empaveli? - store vials of empaveli in the refrigerator between 36°f to 46°f (2°c to 8°c) in the original carton to protect from light. - do not use empaveli past the expiration date stamped on the carton. keep empaveli, empaveli injector, and all medicines out of the reach of children. - find a well-lit, flat work surface area, like a table. - remove a single vial carton of empaveli from the refrigerator. keep the vial in the carton at room temperature and allow it to warm up for about 30 minutes .  do not try to speed up the warming process.  do not try to speed up the warming process. - wash your hands well with soap and water. - dry your hands. - remove the vial from the carton. carefully look at the liquid in the vial of empaveli. - empaveli is a clear, colorless to slightly yellowish liquid. check for particles or color changes.  do not use and call apellisassist if: - flip up to remove the protective flip cap from the top of the vial to show the exposed middle part of the gray rubber stopper of the empaveli vial. - throw away the protective flip cap. - clean the gray rubber stopper on the top of the empaveli vial with a new alcohol wipe. - allow the gray rubber stopper to dry for at least 30 seconds.  do not touch the exposed gray rubber stopper after wiping.  do not remove the needleless transfer device from the blister package.  do not touch the spike or the inside of the needleless transfer device.  do not use the needleless transfer device if it comes out or is dropped out of the package.  do not use the needleless transfer device if the package is opened. - remove the cover of the needleless transfer device package. - place the vial on a clean, flat surface and hold the vial by the base with one hand. - using the outside of the blister package to firmly hold the needleless transfer device, push needleless transfer device straight down onto the vial top until it snaps securely into place. - remove the blister package from the needleless transfer device and throw the blister package away.  do not touch the connector at the top of the transfer device. - remove the syringe from its packaging.  do not touch the tip of the syringe. - attach the syringe to the needleless transfer device by twisting the tip of the syringe to the right (clockwise) onto the top of the needleless transfer device. - turn the empaveli vial upside down. - slowly pull the syringe plunger down to fill the syringe with empaveli. - withdraw all empaveli from the vial into the syringe. - make sure there is 20 ml of empaveli in the syringe. - remove air from syringe by gently pushing on the plunger. - while holding the empaveli vial and syringe, turn the empaveli vial and filled syringe upright and place the bottom of the empaveli vial on a flat surface. - remove the filled syringe from the needleless transfer device with one hand while holding the empaveli vial with the other hand and twisting the filled syringe to the left (counterclockwise). - place the syringe on a clean, flat surface while you prepare the empaveli injector. the syringe will not leak when set down.  do not touch the tip of the filled syringe.  do not remove the needleless transfer device from the vial. - throw away the vial with the needleless transfer device attached into the household trash. important information for administration with empaveli injector - do not use empaveli injector if tamper-proof label has been broken. - do not use empaveli injector if you have a skin condition on your stomach (injection site). - do not use if you dropped empaveli injector. - do not use if the sealed plastic tray is open or damaged. - do not use if the expiration date on the box has passed. - do not use if you have an acrylic allergy. tell your healthcare provider if you are allergic to acrylic. - do not reuse empaveli injector. - do not store the filled empaveli injector. - wear loose clothes so that they do not get in the way of the empaveli injector. - do not store the empaveli injector in direct sunlight. if the empaveli injector is stored in direct sunlight, do not use it and call apellisassist at 1-866-my-apl-assist (1-866-692-7527). - do not apply empaveli injector along the belt line or on areas where the injector will be affected by folds in the skin. - do not touch the white adhesive on the bottom of empaveli injector before attaching to stomach. - do not let your clothes touch the clean site. - do not remove the red safety tab until empaveli injector is attached to body. - do not remove empaveli injector from the skin until the button pops out. - do not throw away (dispose of) the empaveli injector into household trash. see the section "remove and dispose of empaveli injector" for information on how to dispose of the empaveli injector. - choose an injection site at least 1 inch from the edge of your belly button and the edge of the empaveli injector, and 1 inch from last injection site. - use the empaveli injector on stomach only. - do not remove empaveli injector from the skin during injection. - do not bathe, shower, exercise, use hot tubs, whirlpools, or saunas. avoid getting your stomach wet. the empaveli injector is not waterproof. water or sweat may loosen empaveli injector from skin. - do not sleep or bathe during injection. - avoid intense physical activity. - do not bump or knock the empaveli injector. - do not bump the empaveli injector button. - do not use anything to hold the empaveli injector in place. - pick up the syringe filled with empaveli. - twist the filled syringe tip to the right (clockwise) into the fill port until it is tight. - firmly push the syringe plunger down. - the syringe plunger may be hard to push. - watch the fill gauge move as empaveli is pushed into the injector. - make sure the syringe is empty. if needed, press firmly down on the syringe plunger again.  do not remove syringe from filling base. after the empaveli injector is filled, continue with the preparation and injection.  do not store filled empaveli injector. - select an area on your stomach to place the empaveli injector. use the empaveli injector on your stomach only. - choose an injection site at least: 1 inch from the edge of your belly button and the edge of the empaveli injector. and 1 inch from your last injection site. - 1 inch from the edge of your belly button and the edge of the empaveli injector. and - 1 inch from your last injection site.  avoid an injection site that is tender, bruised, red, hard, irritated, scarred, tattooed, or has stretch marks.  do not apply the empaveli injector along the belt line or on areas where the empaveli injector will be affected by folds in the skin.  wear loose clothes so that they do not get in the way of the empaveli injector. - clean the injection site with an alcohol wipe. - allow the injection site to dry for at least 30 seconds.  do not let your clothes touch the clean injection site. - hold the gray pull tab and pull. allow both the gray and clear pull tabs to fall to the side. - both tabs may fall to the side or come off completely.  do not remove the red safety tab until the empaveli injector is attached to the body. - hold the sides of the empaveli injector and pull it straight up to remove it from the filling base.  do not touch the adhesive on the bottom of the empaveli injector or fold the adhesive onto itself. - the white adhesive will stay attached to the empaveli injector and the clear liner will stay attached to the filling base.  do not remove the red safety tab until the empaveli injector is attached to your body. ensure the injection site has been cleaned before attaching empaveli injector. - position the empaveli injector so that the fill window is pointed up toward your face. - press firmly on the clear portion of the empaveli injector to attach to stomach.  do not use anything to hold the empaveli injector in place. - hold the empaveli injector with 1 hand. use the other hand to pull the red safety tab off. - the empaveli injection will not start until the red safety tab is removed. - right away, press the button in firmly until it stays in place to start the empaveli injection. pushing the button in will insert the needle into your skin. you may feel the needle go into your skin. - light daily activities can be done during the empaveli injection.  be careful not to bump or knock the empaveli injector or button during the empaveli injection.  keep your stomach dry. avoid intense physical activity. do not sleep or bathe during your empaveli injection. - your empaveli injection will continue as long as the button is pushed in. it may take approximately 30 to 60 minutes to complete. - to track progress, watch the fill gauge move across fill window toward empty. it may take some time to move and may move slowly.  do not remove the empaveli injector until the button pops out. if the button does not pop out after 2 hours (120 minutes), refer to questions and answers. if the empaveli injector falls off of your body, refer to questions and answers.  caution: holding down the button will stop the flow of medicine. injection will begin again when the button is released. if you have an allergic reaction to the adhesive, call your healthcare provider right away. - when the button pops out, the empaveli injection is done. the needle will be pulled out of the skin and back into the empaveli injector. the button popping out is the only way to know if the empaveli injection is complete. do not remove the empaveli injector until the button pops out. - use your thumb to lift the adhesive tab. hold the adhesive tab against the empaveli injector. - slowly peel the empaveli injector away from your skin. - put your used empaveli injector in an fda-cleared sharps disposal container right away after use.  do not throw away the empaveli injector in the household trash. the filling base with the syringe attached, alcohol wipe, and packaging may be placed 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 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. - made of 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 used needles and syringes. - for more information about safe sharps disposal, and for specific information about sharps disposal in the state that you live in, go to the fda's website at: http:/www.fda.gov/safesharpsdisposal . - do not throw away (dispose of) your used sharps disposal container in your household trash unless your community guidelines permit this. - do not recycle your used sharps disposal container. - keep the used empaveli injector and sharps disposal container out of the reach of children. - keep the empaveli injector in unopened tray inside the original box. - do not open the tray until ready for empaveli injection. - store the empaveli injector unit in clean, dry area away from heat and sunlight, at a temperature between 36°f to 86°f (2°c to 30°c). - use the empaveli injector where the temperature is between 41°f to 104°f (5°c to 40°c).  do not touch the bottom of the empaveli injector as the needle will be exposed. set the empaveli injector aside and call apellisassist at 1-866-my-apl-assist (1-866-692-7527). manufactured for: apellis pharmaceuticals, inc. 100 fifth avenue waltham, ma 02451 manufactured by: enable injections, inc. 2863 e. sharon road cincinnati, oh 45421, usa 10130600 rev 04 patent: enableinjections.com/patent copyright © 2023 apellis pharmaceuticals, inc. all rights reserved. apellis, apellisassist, empaveli, and their respective logos are registered trademarks of apellis pharmaceuticals, inc. this instructions for use has been approved by the u.s. food and drug administration. issued: 09/2023 emp inj-ifu-29sep2023-1.0

الاتحاد الأوروبي - الإنجليزية - EMA (European Medicines Agency)

alexion europe sas - ravulizumab - hemoglobinuria, paroxysmal - selective immunosuppressants - paroxysmal nocturnal haemoglobinuria (pnh)ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with pnh:- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months (see section 5.1).atypical haemolytic uremic syndrome (ahus)ultomiris is indicated in the treatment of patients with a body weight of 10 kg or above with ahus who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab (see section 5.1).generalized myasthenia gravis (gmg)ultomiris is indicated as an add-on to standard therapy for the treatment of adult patients with gmg who are anti-acetylcholine receptor (achr) antibody-positive.neuromyelitis optica spectrum disorder (nmosd)ultomiris is indicated in the treatment of adult patients with nmosd who are anti-aquaporin 4 (aqp4) antibody-positive (see section 5.1).ultomiris is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (pnh):- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months.ultomiris is indicated in the treatment of adult patients with atypical haemolytic uremic syndrome (ahus) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.

الولايات المتحدة - الإنجليزية - 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 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 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, and 54 with gmg) 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).

إسرائيل - الإنجليزية - Ministry of Health

alexion pharma israel ltd - ravulizumab - concentrate for solution for infusion - ravulizumab 10 mg/ml - ravulizumab - ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with paroxysmal nocturnal haemoglobinuria (pnh): • in patients with haemolysis with clinical symptom(s) indicative of high disease activity. • in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months. ultomiris is indicated in the treatment of patients with a body weight of 10 kg or above with atypical haemolytic uremic syndrome (ahus) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.ultomiris is indicated in the treatment of adult patients with generalized myasthenia gravis (gmg) who are anti-acetylcholine receptor (achr) antibody-positive.

الولايات المتحدة - الإنجليزية - 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)] .

الولايات المتحدة - الإنجليزية - NLM (National Library of Medicine)

wyeth pharmaceutical division of wyeth holdings llc - neisseria meningitidis group b recombinant lp2086 a05 protein variant antigen (unii: 583wcd0izi) (neisseria meningitidis group b recombinant lp2086 a05 protein variant antigen - unii:583wcd0izi), neisseria meningitidis group b recombinant lp2086 b01 protein variant antigen (unii: 7mbd4k530d) (neisseria meningitidis group b recombinant lp2086 b01 protein variant antigen - unii:7mbd4k530d) - neisseria meningitidis group b recombinant lp2086 a05 protein variant antigen 60 ug in 0.5 ml - trumenba is indicated for active immunization to prevent invasive disease caused by neisseria meningitidis serogroup b. trumenba is approved for use in individuals 10 through 25 years of age. severe allergic reaction (e.g. anaphylaxis) to any component of trumenba [see description (11)] . risk summary all pregnancies have a 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% to 4% and 15% to 20%, respectively. there are no adequate and well-controlled studies of trumenba in pregnant women. available human data on trumenba administered to pregnant women are insufficient to inform vaccine-associated risks in pregnancy. two developmental toxicity studies were performed in female rabbits administered trumenba prior to mating and during gestation. the dose was 0.5 ml at each occasion (a single human dose is 0.5 ml). these studies revealed no evidence of harm to the fet

الولايات المتحدة - الإنجليزية - NLM (National Library of Medicine)

sanofi pasteur inc. - neisseria meningitidis group a capsular polysaccharide diphtheria toxoid conjugate antigen (unii: re9a0h8oab) (neisseria meningitidis group a capsular polysaccharide diphtheria toxoid conjugate antigen - unii:re9a0h8oab), neisseria meningitidis group c capsular polysaccharide diphtheria toxoid conjugate antigen (unii: 2j57k2523t) (neisseria meningitidis group c capsular polysaccharide diphtheria toxoid conjugate antigen - unii:2j57k2523t), neisseria meningitidis group y capsular polysaccharide diphtheria - neisseria meningitidis group a capsular polysaccharide diphtheria toxoid conjugate antigen 4 ug in 0.5 ml - menactra® , meningococcal (groups a, c, y and w-135) polysaccharide diphtheria toxoid conjugate vaccine, is indicated for active immunization to prevent invasive meningococcal disease caused by neisseria meningitidis serogroups a, c, y and w-135. menactra is approved for use in individuals 9 months through 55 years of age. menactra does not prevent n meningitidis serogroup b disease. severe allergic reaction (eg, anaphylaxis) after a previous dose of a meningococcal capsular polysaccharide-, diphtheria toxoid- or crm197 -containing vaccine, or to any component of menactra [see description (11) ]. pregnancy exposure registry there is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to menactra during pregnancy. to enroll in or obtain information about the registry, call sanofi pasteur at 1-800-822-2463. risk summary all pregnancies have a risk of birth defect, loss, or other adverse outcomes. in the us general population, the estimated background risk of major birth defects an

الولايات المتحدة - الإنجليزية - NLM (National Library of Medicine)

glaxosmithkline biologicals sa - neisseria meningitidis group b nhba fusion protein antigen (unii: 28e911y7ae) (neisseria meningitidis group b nhba fusion protein antigen - unii:28e911y7ae), neisseria meningitidis group b fhbp fusion protein antigen (unii: 25db599g64) (neisseria meningitidis group b fhbp fusion protein antigen - unii:25db599g64), neisseria meningitidis group b nada protein antigen (unii: 1s25r442rs) (neisseria meningitidis group b nada protein antigen - unii:1s25r442rs), neisseria meningitidis group b strain nz98/254 outer membrane vesicle antigen (unii: 91523m4s24) (neisseria meningitidis group b strain nz98/254 outer membrane vesicle antigen - unii:91523m4s24) - neisseria meningitidis serogroup b nhba fusion protein antigen 50 ug in 0.5 ml - bexsero is a vaccine indicated for active immunization to prevent invasive disease caused by neisseria meningitidis serogroup b. bexsero is approved for use in individuals aged 10 through 25 years. approval of bexsero is based on demonstration of immune response, as measured by serum bactericidal activity against three serogroup b strains representative of prevalent strains in the united states. the effectiveness of bexsero against diverse serogroup b strains has not been confirmed. hypersensitivity, including severe allergic reaction, to any component of the vaccine, or after a previous dose of bexsero [see description (11)] . risk summary all pregnancies have a 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% to 4% and 15% to 20%, respectively. there are no adequate and well-controlled studies of bexsero in pregnant women in the u.s. available human data on bexsero administered to pregnant women are insufficient to inform vaccine-associated risks in pregnancy. a developmental toxicity study was performed in female rabbits administered bexsero prior to mating and during gestation. the dose was 0.5 ml at each occasion (a single human dose is 0.5 ml). this study revealed no adverse effects on fetal or pre-weaning development due to bexsero (see data) . data animal data: in a developmental toxicity study, female rabbits were administered bexsero by intramuscular injection on days 29, 15, and 1 prior to mating and on gestation days 7 and 20. the total dose was 0.5 ml at each occasion (a single human dose is 0.5 ml). no adverse effects on pre-weaning development up to postnatal day 29 were observed. there were no fetal malformations or variations observed. risk summary it is not known whether the vaccine components of bexsero are excreted in human milk. available data are not sufficient to assess the effects of bexsero on the breastfed infant or on milk production/excretion. the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for bexsero and any potential adverse effects on the breastfed child from bexsero or from the underlying maternal condition. for preventive vaccines, the underlying maternal condition is susceptibility to disease prevented by the vaccine. safety and effectiveness of bexsero have not been established in children younger than 10 years. safety and effectiveness of bexsero have not been established in adults older than 65 years.