{"id":182,"date":"2021-08-03T11:26:26","date_gmt":"2021-08-03T11:26:26","guid":{"rendered":"https:\/\/medinaz.com\/blog\/?p=182"},"modified":"2022-08-08T21:30:53","modified_gmt":"2022-08-08T21:30:53","slug":"zolgensma-worlds-most-expensive-drug","status":"publish","type":"post","link":"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/","title":{"rendered":"Zolgensma: World&#8217;s Most Expensive Drug for SMA &#8211; All you need to know"},"content":{"rendered":"\n<p>Zolgensma World&#8217;s Most Expensive Drug is used for Spinal Muscular Atrophy (SMA)<\/p>\n\n\n\n<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_55 counter-hierarchy ez-toc-counter ez-toc-grey ez-toc-container-direction\">\n<p class=\"ez-toc-title\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-69e021ee739ee\"><span class=\"\"><span style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-69e021ee739ee\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#What_is_Zolgensma\" title=\"What is Zolgensma ?\">What is Zolgensma ?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#What_is_spinal_muscular_atrophy_SMA\" title=\"What is spinal muscular atrophy (SMA)?\">What is spinal muscular atrophy (SMA)?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#Zolgensma_mechanism_of_action_How_it_works\" title=\"Zolgensma mechanism of action (How it works)\">Zolgensma mechanism of action (How it works)<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#Zolgensma_Dosage\" title=\"Zolgensma Dosage:\">Zolgensma Dosage:<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#What_are_the_side_effects_of_Zolgensma\" title=\"What are the side effects of Zolgensma?\">What are the side effects of Zolgensma?<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#Boxed_Warning\" title=\"Boxed Warning:\">Boxed Warning:<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-7\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#Zolgensma_World%E2%80%99s_Most_Expensive_Drug_Price\" title=\"Zolgensma: World&#8217;s Most Expensive Drug Price\">Zolgensma: World&#8217;s Most Expensive Drug Price<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-8\" href=\"https:\/\/medinaz.com\/blog\/2021\/08\/03\/zolgensma-worlds-most-expensive-drug\/#Insurance_coverage\" title=\"Insurance coverage\">Insurance coverage<\/a><\/li><\/ul><\/nav><\/div>\n<h2><span class=\"ez-toc-section\" id=\"What_is_Zolgensma\"><\/span>What is Zolgensma ?<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Zolgensma (Onasemnogene abeparvovec) is an adeno-associated virus vector-based gene therapy that delivers a fully functional copy of human SMN gene into the target motor neuron cells.<\/p>\n\n\n\n<p>Zolgensma contains the active substance called onasemnogene abeparvovec.<\/p>\n\n\n\n<p>The U.S. Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA)<\/p>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"What_is_spinal_muscular_atrophy_SMA\"><\/span>What is spinal muscular atrophy (SMA)?<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>SMA is a rare genetic disease caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the survival motor neuron (SMN) protein \u2013 a protein found throughout the body, which is critical for the maintenance and function of specialized nerve cells, called motor neurons.<\/p>\n\n\n\n<p>Motor neurons in the brain and spinal cord control muscle movement throughout the body. If there is not enough functional SMN protein, then the motor neurons die, leading to debilitating and often fatal muscle weakness.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" width=\"1024\" height=\"576\" src=\"https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector2-1024x576.jpg\" alt=\"Zolgensma: World's Most Expensive Drug \" class=\"wp-image-184\" srcset=\"https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector2-1024x576.jpg 1024w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector2-300x169.jpg 300w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector2-768x432.jpg 768w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector2-1536x864.jpg 1536w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector2-800x450.jpg 800w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption>Normal &amp; Abnormal Nerves and Muscles<\/figcaption><\/figure>\n\n\n\n<p>Children with this condition have problems holding their head up, swallowing and breathing. These symptoms may be present at birth or may present by the age of 6 months<\/p>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"Zolgensma_mechanism_of_action_How_it_works\"><\/span>Zolgensma mechanism of action (How it works)<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Zolgensma (Onasemnogene abeparvovec) is an adeno-associated virus vector-based gene therapy that delivers a fully functional copy of human SMN gene into the target motor neuron cells. A one-time intravenous administration results in expression of the SMN protein in a child&#8217;s motor neurons, which improves muscle movement and function and survival of a child with SMA<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" width=\"1024\" height=\"576\" src=\"https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector-1024x576.jpg\" alt=\"Zolgensma mechanism of action\" class=\"wp-image-185\" srcset=\"https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector-1024x576.jpg 1024w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector-300x169.jpg 300w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector-768x432.jpg 768w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector-1536x864.jpg 1536w, https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Adenovector-800x450.jpg 800w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption>Zolgensma mechanism of action<\/figcaption><\/figure>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"Zolgensma_Dosage\"><\/span>Zolgensma Dosage:<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Only applicable for children &lt;2 years old<\/p>\n\n\n\n<p>Give as a slow IV infusion over 60mins (1.1\u00d710<sup>14<\/sup>&nbsp;vg\/kg ). Starting one day prior to Zolgensma infusion: give systemic corticosteroids equivalent to oral prednisolone 1mg\/kg\/day for 30 days, then taper dose for the next 28 days if LFTs are unremarkable<\/p>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"What_are_the_side_effects_of_Zolgensma\"><\/span>What are the side effects of Zolgensma?<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>The most common side effects of Zolgensma are <strong><em>elevated liver enzymes<\/em><\/strong> and <strong><em>vomiting<\/em><\/strong><\/p>\n\n\n\n<p>Other possible side effects &#8211; Elevated aminotransferases, thrombocytopenia, elevated troponin-I.<\/p>\n\n\n\n<p>Rare side effects:<\/p>\n\n\n\n<ul><li>Decreased Blood Platelets<\/li><li>Leukoencephalopathy, A Disease Of The White Matter Of The Brain<\/li><li>Lung Failure Causing Loss Of Breath<\/li><\/ul>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"Boxed_Warning\"><\/span>Boxed Warning:<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Zolgensma has a boxed warning that <strong><em>acute serious liver injury<\/em><\/strong> can occur.<\/p>\n\n\n\n<p>Patients with pre existing liver impairment may be at higher risk of experiencing serious liver injury.<\/p>\n\n\n\n<p>Clinical examination and laboratory tests to assess liver function should be completed prior to treatment with Zolgensma, and patients\u2019 liver function should be monitored for at least <strong><em>three months after<\/em><\/strong> Zolgensma administration<\/p>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"Zolgensma_World%E2%80%99s_Most_Expensive_Drug_Price\"><\/span>Zolgensma: World&#8217;s Most Expensive Drug Price<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>It is a one-time-only gene therapy that costs <strong><em>$2.1 million<\/em><\/strong> for the <strong><em>single<\/em><\/strong> treatment. Currently it is the world\u2019s most expensive drug.<\/p>\n\n\n\n<h2><span class=\"ez-toc-section\" id=\"Insurance_coverage\"><\/span><strong>Insurance coverage<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Zolgensma is so expensive because it is used for the treatment of a rare disease SMA. The expertise, time and investment required to make it and the research has taken very long. The market size for manufacturing this drug is also very limited and small.<\/p>\n\n\n\n<p>Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn\u2019t work.<\/p>\n\n\n\n<p>Novartis has decided that the worth of this drug is calculated basis how many years of life is gained as well as taking into account the effectiveness of the drug. They claim that this drug dramatically transforms the lives of families affected by this devastating disease.<\/p>\n\n\n\n<p>As it is a one-time treatment for SMA, many health insurances do not allow this at such a big price tag and also there is no guarantee that a drug will meet all expectations.&nbsp;<\/p>\n\n\n\n<p><\/p>\n\n\n\n<p>Reference:<\/p>\n\n\n\n<p><a href=\"https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease\">https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease<\/a><\/p>\n\n\n\n<p><a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC6652281\/\">https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC6652281\/<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Zolgensma World&#8217;s Most Expensive Drug is used for Spinal Muscular Atrophy (SMA) What is Zolgensma ? Zolgensma (Onasemnogene abeparvovec) is an adeno-associated virus vector-based gene therapy that delivers a fully functional copy of human SMN gene into the target motor neuron cells. Zolgensma contains the active substance called onasemnogene abeparvovec. The U.S. Food and Drug<\/p>\n","protected":false},"author":1,"featured_media":183,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"ub_ctt_via":""},"categories":[3],"tags":[57,56],"featured_image_src":"https:\/\/medinaz.com\/blog\/wp-content\/uploads\/2021\/08\/Zolgensma-Worlds-Most-Expensive-Drug.png","author_info":{"display_name":"Medinaz Academy","author_link":"https:\/\/medinaz.com\/blog\/author\/medinaz-blog-admin\/"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v19.4 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Zolgensma: World&#039;s Most Expensive Drug for SMA - All you need to know - Medinaz Blog<\/title>\n<meta name=\"description\" content=\"Zolgensma World&#039;s Most Expensive Drug cost around $ 2.1 million (16 crore rupees ) per dose. Why is Zolgensma so expensive? 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Why is Zolgensma so expensive? 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