{"id":36464,"date":"2022-11-28T07:50:58","date_gmt":"2022-11-28T07:50:58","guid":{"rendered":"https:\/\/zeepedia.com\/?p=36464"},"modified":"2022-11-29T10:16:12","modified_gmt":"2022-11-29T10:16:12","slug":"zolgensma","status":"publish","type":"post","link":"https:\/\/zeepedia.com\/medical\/medicines\/zolgensma\/","title":{"rendered":"Zolgensma &#8211; Onasemnogen-abeparvovek uses, dose and side effects"},"content":{"rendered":"\n<p>2 \u00d7 10\u00a0<sup>13<\/sup>\u00a0\u00a0vector genome\/ml\u00a0infusion fluid\u00a0,solution<br>caseinogen-abeparvovek<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">What Zolgensma is and what it is used for<\/h2>\n\n\n\n<p><strong>What Zolgensma is<\/strong><\/p>\n\n\n\n<p>Zolgensma is a type of medicine called gene therapy.\u00a0It contains the active substance caseinogen-abeparvovek, which contains human genetic material.<\/p>\n\n\n\n<p><strong>What Zolgensma is used for<\/strong><\/p>\n\n\n\n<p>Zolgensma is used to treat &#8216;spinal muscular atrophy (SMA), a rare, serious inherited disease.<\/p>\n\n\n\n<p><strong>How Zolgensma works<\/strong><\/p>\n\n\n\n<p>SMA is caused by a lack of an abnormal version of a gene needed to make a necessary\u00a0protein\u00a0called survival motor neuron (SMN).\u00a0The absence of SMN\u00a0protein\u00a0means that nerves that control muscles (motor neurons) die.\u00a0This leads to the muscles becoming weak and atrophied and ultimately to loss of mobility.<\/p>\n\n\n\n<p>This medicine works by providing a fully functional copy of the SMN gene which then helps the body produce enough SMN\u00a0protein.\u00a0The genes are delivered into the cells where they are needed using a modified\u00a0virus\u00a0that does not cause disease in humans.<br><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">What you need to know before your child receives Zolgensma<\/h2>\n\n\n\n<h2 class=\"wp-block-heading\">Do NOT use Zolgensma<\/h2>\n\n\n\n<ul class=\"wp-block-list\"><li>if your child is allergic to caseinogen-abeparvovek or any of the other ingredients of this medicine (listed in section 6).<\/li><\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Warnings and precautions<\/h2>\n\n\n\n<p>Your child&#8217;s doctor will test for&nbsp;antibodies&nbsp;before treatment to determine if this medicine is suitable for your child.<\/p>\n\n\n\n<p>Liver problems<\/p>\n\n\n\n<p>Talk to your child&#8217;s doctor or nurse before giving this medicine if your child has had any liver problems.\u00a0This medicine can lead to an increase in the\u00a0enzymes\u00a0(\u00a0proteins\u00a0found in the body) produced in the liver or damage to the liver.\u00a0Possible signs to look out for after your child has been given this medicine include vomiting, jaundice (yellowing of the skin or the whites of the eyes), or decreased alertness (see section 4 for more information).<\/p>\n\n\n\n<p>Your child will have a blood test to check how well the liver is working before starting treatment with Zolgensma.&nbsp;Your child will also have regular blood tests at least 3 months after treatment to check for increases in liver enzymes.<\/p>\n\n\n\n<p>Infection<\/p>\n\n\n\n<p>An\u00a0infection\u00a0(eg cold,\u00a0flu,\u00a0or bronchiolitis) before or after Zolgensma treatment can lead to more serious complications.\u00a0You must pay attention to signs of an\u00a0infection\u00a0such as\u00a0cough, wheezing, sneezing, runny nose, sore throat, or fever.\u00a0Tell your child&#8217;s doctor immediately if you notice that your child develops any of these symptoms.<\/p>\n\n\n\n<p>Regular blood tests<\/p>\n\n\n\n<p>This medicine can reduce the number of&nbsp;blood platelets&nbsp;(&nbsp;thrombocytopenia&nbsp;).&nbsp;You need to be aware of any signs of low&nbsp;platelet count&nbsp;after your child has received Zolgensma such as&nbsp;abnormal bruising or bleeding (see section 4 for more information).&nbsp;Most of the cases of low&nbsp;platelet counts&nbsp;that have been reported occurred within two weeks of the child receiving Zolgensma.<\/p>\n\n\n\n<p>Zolgensma can increase the levels of a heart protein called &#8220;troponin I&#8221; and maybe a sign of damage to the heart.\u00a0You need to watch out for any signs of heart problems after your child has been given this medicine, such as\u00a0pale gray or blue skin color, difficulty breathing, and swelling of the arms and legs or stomach (see section 4 for more information).<\/p>\n\n\n\n<p>Before starting treatment with Zolgensma, your child will have a blood test to check the number of\u00a0blood cells\u00a0(including\u00a0red blood cells\u00a0and\u00a0platelets\u00a0) and the troponin I level in the body.\u00a0They will also have blood tests to check the creatinine level, which is a measure of how well the kidneys are working. Sometime after treatment, your child will also have regular blood tests to check changes in\u00a0platelets\u00a0and troponin I levels.<\/p>\n\n\n\n<p>Abnormal clotting of blood in small blood vessels (thrombotic microangiopathy)<\/p>\n\n\n\n<p>There have been reports of patients developing thrombotic microangiopathy approximately one week after Zolgensma treatment.\u00a0Thrombotic microangiopathy is accompanied by a decrease in\u00a0red blood cells\u00a0and cells involved in\u00a0coagulation\u00a0(\u00a0platelets\u00a0).\u00a0These blood clots can affect your baby&#8217;s kidneys.\u00a0Your child&#8217;s doctor may want to check your child&#8217;s blood (\u00a0platelet count\u00a0) and\u00a0blood pressure.\u00a0Possible signs to look out for after your child has received Zolgensma include bruising easily, convulsions (seizures), or a decrease in the amount of urine (see section 4 for more information).\u00a0Contact a doctor immediately if your child develops any of these signs.<\/p>\n\n\n\n<p>Blood, organ,\u00a0tissue,\u00a0and cell donation<\/p>\n\n\n\n<p>After your child has been treated with Zolgensma, he\/she will not be able to donate blood, organs,\u00a0tissue,\u00a0or cells.\u00a0This is because Zolgensma is a gene therapy medicine.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Other medicines and Zolgensma<\/h2>\n\n\n\n<p>Tell your child&#8217;s doctor or nurse if your child is taking, has recently taken, or might be taking other medicines.<\/p>\n\n\n\n<p>Prednisolone<\/p>\n\n\n\n<p>For about 2 months or longer, your child will also receive a corticosteroid medicine eg.&nbsp;prednisolone&nbsp;(see also section 3) as part of Zolgensma treatment.&nbsp;The corticosteroid medicine helps treat increases in liver enzymes that your child may develop after receiving Zolgensma.<\/p>\n\n\n\n<p>Vaccinations<\/p>\n\n\n\n<p>Because&nbsp;corticosteroids&nbsp;can affect the body&#8217;s immune system (defense system)<\/p>\n\n\n\n<p><strong>your child&#8217;s doctor may decide to delay certain vaccinations<\/strong><\/p>\n\n\n\n<p>during the time the child receives corticosteroid treatment.&nbsp;Talk to your child&#8217;s doctor or nurse if you have any questions.<a><\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Zolgensma contains sodium<\/h2>\n\n\n\n<p>This medicinal product contains 4.6 mg of\u00a0sodium\u00a0per ml corresponding to 0.23% of the WHO maximum recommended daily intake (2 grams of\u00a0sodium\u00a0for adults).\u00a0Each 5.5 ml\u00a0vial\u00a0contains 25.3 mg\u00a0sodium\u00a0and each 8.3 ml\u00a0vial\u00a0contains 38.2 mg\u00a0sodium.<\/p>\n\n\n\n<p><strong>Additional information for parents\/caregivers<\/strong><\/p>\n\n\n\n<p>Advanced SMA<\/p>\n\n\n\n<p>Zolgensma can save living motor neurons but does not save dead motor neurons.\u00a0Children with less severe symptoms of SMA (such as lack of reflexes or decreased\u00a0muscle tone\u00a0) may have enough viable motor neurons to benefit significantly from treatment with Zolgensma.\u00a0Zolgensma may not work as well in children with severe muscle weakness or paralysis, breathing problems or in children who cannot swallow, or children who have extensive malformations (eg heart defects), including patients with SMA type 0, as it may mean limited to improvement after treatment with Zolgensma.\u00a0Your child&#8217;s doctor will decide whether your child should receive this medicine.<\/p>\n\n\n\n<p>Hygiene care<\/p>\n\n\n\n<p>The active substance in Zolgensma may be temporarily excreted via bodily fluids and feces from your child;\u00a0this is called &#8220;excretion&#8221;.\u00a0Parents and caregivers must observe good hand hygiene for up to 1 month after the child has received Zolgensma.\u00a0Use protective gloves when in direct contact with the child&#8217;s body fluids or feces and wash your hands thoroughly afterward with soap and running warm water or an alcohol-based hand sanitizer.\u00a0Double bags should be used to dispose of soiled diapers and other waste.\u00a0Disposable diapers can still be thrown into household waste.<\/p>\n\n\n\n<p>You should continue to follow these instructions for at least 1 month after your child&#8217;s treatment with Zolgensma.&nbsp;Talk to your child&#8217;s doctor or nurse if you have any questions.<a><\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">How to use Zolgensma<\/h2>\n\n\n\n<p>Zolgensma is given by a doctor or nurse trained in treating your child&#8217;s condition.<\/p>\n\n\n\n<p>The doctor will calculate the amount of Zolgensma your child should receive according to the child&#8217;s weight.&nbsp;Zolgensma is given&nbsp;intravenously&nbsp;(into a vein) as a single infusion (drip) over about 1 hour.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Zolgensma is only given ONCE to your child.<\/h2>\n\n\n\n<p>Your child will also receive&nbsp;prednisolone&nbsp;(or another&nbsp;corticosteroid&nbsp;) by mouth starting 24 hours before he\/she receives Zolgensma.&nbsp;The dose&nbsp;of a&nbsp;corticosteroid&nbsp;also depends on the child&#8217;s weight.&nbsp;Your child&#8217;s doctor will work out the total&nbsp;dose&nbsp;to be given.<\/p>\n\n\n\n<p>Your child will receive corticosteroid therapy daily for about 2 months after&nbsp;dose&nbsp;one of Zolgensma, or until the child&#8217;s liver enzymes decrease to an acceptable level.&nbsp;The doctor will reduce the corticosteroid dose slowly until treatment can be stopped completely.<\/p>\n\n\n\n<p>If you have any further questions, contact your child&#8217;s doctor or nurse.<a><\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Possible side effects<\/h2>\n\n\n\n<p>Like all medicines, this medicine can cause\u00a0side effects, although not everybody gets them.<\/p>\n\n\n\n<p><strong>Contact a doctor immediately<\/strong>&nbsp;if your child develops any of the following serious side effects:<\/p>\n\n\n\n<p><strong>Common<\/strong>&nbsp;(may affect up to 1 in 10 users):<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li>bruising or bleeding that lasts longer than usual if the child has been ill &#8211; these may be signs of low\u00a0platelet counts<\/li><li>pale gray or blue skin color, breathing problems (eg rapid breathing, shortness of breath), swelling of the arms and legs or stomach &#8211; these may be signs of possible problems with the heart.<\/li><\/ul>\n\n\n\n<p><strong>Has been reported<\/strong>&nbsp;(occurring in an unknown number of users):<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li>vomiting, jaundice (yellowing of the skin or the whites of the eyes), or decreased alertness &#8211; these may be signs of liver damage<\/li><li>bruising easily, convulsions (seizures), or a decrease in the amount of urine \u2013 these may be signs of thrombotic microangiopathy.<\/li><\/ul>\n\n\n\n<p>Talk to your child&#8217;s doctor or nurse if the child develops other\u00a0side effects.\u00a0These may include:<\/p>\n\n\n\n<p><strong>Very common<\/strong>&nbsp;(may affect more than 1 in 10 users):<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li>increases in liver enzymes seen in blood tests.<\/li><\/ul>\n\n\n\n<p><strong>Common<\/strong>&nbsp;(may affect up to 1 in 10 users):<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li>vomiting<\/li><li>fever.<\/li><\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">How to store Zolgensma<\/h2>\n\n\n\n<p>Keep this medicine out of the sight and reach of children.<\/p>\n\n\n\n<p>The following information is intended for healthcare professionals who will prepare and administer the medicine.<\/p>\n\n\n\n<p>Do not use before the expiry date which is stated on the vial and carton and the label after EXP.\u00a0The expiration date is the last day of the specified month.<\/p>\n\n\n\n<p>Vials must be transported deep-frozen (at or below \u221260 \u00b0C).<\/p>\n\n\n\n<p>Upon receipt, vials should be immediately refrigerated at 2\u00b0C to 8\u00b0C and stored in the original carton.&nbsp;Treatment with Zolgensma must be started within 14 days of receiving the vials.<\/p>\n\n\n\n<p>This medicine contains genetically modified\u00a0organisms.\u00a0Unused medicine or waste must be disposed of according to local guidelines for handling biological waste.\u00a0As this medicine will be given by a doctor, the doctor is responsible for the proper disposal of the product.\u00a0These measures will help to protect the environment.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Contents of the packaging and other information<\/h2>\n\n\n\n<h2 class=\"wp-block-heading\">Contents declaration<\/h2>\n\n\n\n<ul class=\"wp-block-list\"><li>The active substance is onasemnogen-abeparvovek.\u00a0Each\u00a0vial\u00a0contains caseinogen-apeparvovek at a nominal concentration of 2 \u00d7 10\u00a013\u00a0\u00a0vector genomes\/ml.<\/li><li>Other ingredients are tromethamine, magnesium chloride, sodium chloride, poloxamer 188\u00a0hydrochloric acid\u00a0(for\u00a0pH\u00a0adjustment), and water for injections.<\/li><\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Appearance and package sizes of the medicine<\/h2>\n\n\n\n<p>Zolgensma is a clear to slightly opaque, colorless to slightly whitish solution for\u00a0infusion.<\/p>\n\n\n\n<p>Zolgensma is supplied in vials containing a nominal fill volume of either 5.5 ml or 8.3 ml.&nbsp;Each&nbsp;vial&nbsp;is for single use only.<\/p>\n\n\n\n<p>Each carton contains between 2 and 14 vials.<a><\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Marketing authorization holder and manufacturer<\/h2>\n\n\n\n<p>Novartis Europharm Limited<\/p>\n\n\n\n<p>Vista Building<\/p>\n\n\n\n<p>Elm Park, Merrion Road<\/p>\n\n\n\n<p>Dublin 4<\/p>\n\n\n\n<p>Ireland<\/p>\n\n\n\n<p><strong>Manufacturer<\/strong><\/p>\n\n\n\n<p>Almac Pharma Services Limited<\/p>\n\n\n\n<p>Finnabair Industrial Estate<\/p>\n\n\n\n<p>Dundalk, Co.&nbsp;Louth<\/p>\n\n\n\n<p>A91 P9KD<\/p>\n\n\n\n<p>Ireland<\/p>\n\n\n\n<p>Contact the representative of the Marketing Authorization Holder if you would like to know more about this medicine:<\/p>\n\n\n\n<figure class=\"wp-block-table\"><table><tbody><tr><td><strong>Belgi\u00eb\/Belgique\/Belgien<\/strong>Novartis Pharma NVT\u00e9l\/Tel: +32 2 246 16 11<\/td><td><strong>Lithuania<\/strong>SIA Novartis Baltics Lietuvos filialasPhone: +370 5 269 16 50<\/td><\/tr><tr><td><strong>Bulgaria<\/strong>Novartis Bulgaria EOODPhone: +359 2 489 98 28<\/td><td><strong>Luxembourg\/Luxembourg<\/strong>Novartis Pharma NVT\u00e9l\/Tel: +32 2 246 16 11<\/td><\/tr><tr><td><strong>Czech Republic<\/strong>Novartis s.r.oPhone: +420 225 775 111<\/td><td><strong>Magyarorsz\u00e1g<\/strong>Novartis Hung\u00e1ria Kft.Tel.: +36 1 457 65 00<\/td><\/tr><tr><td><strong>Denmark<\/strong>Novartis Healthcare A\/SPhone: +45 39 16 84 00<\/td><td><strong>Malta<\/strong>Novartis Pharma Services Inc.Phone: +356 2122 2872<\/td><\/tr><tr><td><strong>Deutschland<\/strong>Novartis Pharma GmbHPhone: +49 911 273 0<\/td><td><strong>The Netherlands<\/strong>Novartis Pharma BVPhone: +31 88 04 52 111<\/td><\/tr><tr><td><strong>Estonia<\/strong>SIA Novartis Baltics Eesti branch phone: +372 66 30 810<\/td><td><strong>Norway<\/strong>Novartis Norway iPhone: +47 23 05 20 00<\/td><\/tr><tr><td><strong>Greece<\/strong>Novartis (Greece) AEBEPhone: +30 210 281 17 12<\/td><td><strong>Austria<\/strong>Novartis Pharma GmbHPhone: +43 1 86 6570<\/td><\/tr><tr><td><strong>Spain<\/strong>Novartis Farmac\u00e9utica, SAPhone: +34 93 306 42 00<\/td><td><strong>Polish<\/strong>Novartis Poland Sp.\u00a0z o. iPhone: +48 22 375 4888<\/td><\/tr><tr><td><strong>France<\/strong>Novartis Pharma SASTel: +33 1 55 47 66 00<\/td><td><strong>Portugal<\/strong>Novartis Farma &#8211; Produtos Farmac\u00eauticos, SAPhone: +351 21 000 8600<\/td><\/tr><tr><td><strong>Hrvatska<\/strong>Novartis Hrvatska dooTel.&nbsp;+385 1 6274 220<\/td><td><strong>Romania<\/strong>Novartis Pharma Services Romania SRLPhone: +40 21 31299 01<\/td><\/tr><tr><td><strong>Ireland<\/strong>Novartis Ireland LimitedPhone: +353 1 260 12 55<\/td><td><strong>Slovenija<\/strong>Novartis Pharma Services Inc.Phone: +386 1 300 75 50<\/td><\/tr><tr><td><strong>Iceland<\/strong>Vistor hf.Phone: +354 535 7000<\/td><td><strong>Slovenian Republic<\/strong>Novartis Slovakia s.r.oPhone: +421 2 5542 5439<\/td><\/tr><tr><td><strong>Italy<\/strong>Novartis Pharma SpAPhone: +39 02 96 54 1<\/td><td><strong>Finland\/Finland<\/strong>Novartis Finland OyTel: +358 (0)10 6133 200<\/td><\/tr><tr><td><strong>Cyprus<\/strong>Novartis Pharma Services Inc.Phone: +357 22 690 690<\/td><td><strong>Sweden<\/strong>Novartis Sweden ABPhone: +46 8 732 32 00<\/td><\/tr><tr><td><strong>Latvia<\/strong>SIA Novartis BalticsPhone: +371 67 887 070<\/td><td><strong>United Kingdom (Northern Ireland)<\/strong>Novartis Ireland LimitedPhone: +44 1276 698370<\/td><\/tr><\/tbody><\/table><\/figure>\n","protected":false},"excerpt":{"rendered":"<p>Zolgensma is a type of medicine called gene therapy.\u00a0It contains the active substance onasemnogen-abeparvovek, which contains human genetic material.<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"cybocfi_hide_featured_image":"","footnotes":""},"categories":[2200,22506],"tags":[28509,28514,28506,28508,28507,28512,28515,28511,28510,28513],"class_list":["post-36464","post","type-post","status-publish","format-standard","hentry","category-medicines","category-z","tag-benefits-of-onasemnogen-abeparvovek","tag-benefits-of-zolgensma","tag-onasemnogen-abeparvovek","tag-onasemnogen-abeparvovek-side-effects","tag-what-is-onasemnogen-abeparvovek","tag-what-is-zolgensma","tag-what-onasemnogen-abeparvovek-is-used-for","tag-what-zolgensma-is-used-for","tag-zolgensma","tag-zolgensma-side-effects"],"_links":{"self":[{"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/posts\/36464","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/comments?post=36464"}],"version-history":[{"count":3,"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/posts\/36464\/revisions"}],"predecessor-version":[{"id":36656,"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/posts\/36464\/revisions\/36656"}],"wp:attachment":[{"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/media?parent=36464"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/categories?post=36464"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/zeepedia.com\/medical\/wp-json\/wp\/v2\/tags?post=36464"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}