{"id":16124,"date":"2019-11-26T22:43:51","date_gmt":"2019-11-26T21:43:51","guid":{"rendered":"https:\/\/www.hollandbio.nl\/?p=16124"},"modified":"2019-11-26T22:43:51","modified_gmt":"2019-11-26T21:43:51","slug":"alnylams-givlaari-receives-fda-approval","status":"publish","type":"post","link":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/","title":{"rendered":"Alnylam\u2019s GIVLAARI receives FDA approval"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">Alnylam has received FDA approval for GIVLAARI<sup>\u2122<\/sup>&nbsp;(givosiran) for the treatment of adults with acute hepatic\nporphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized\nby debilitating, potentially life-threatening attacks and, for some patients,\nchronic manifestations that negatively impact daily functioning and quality of\nlife. Long-term complications of AHP can include chronic neuropathic pain,\nhypertension, chronic kidney disease and liver disease. GIVLAARI was shown to\nsignificantly reduce the rate of porphyria attacks that required\nhospitalizations, urgent healthcare visits or IV hemin administration at home.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">&nbsp;\u201cWe believe the approval of GIVLAARI\nrepresents a landmark event for the advancement of precision genetic medicines,\nproviding new hope for patients and their caregivers living with the\ndebilitating manifestations of AHP and unpredictable nature of AHP attacks, as\nwell as for the doctors who diagnose and treat these patients. We are grateful\nto the investigators, patients and families who have helped make this new\ntreatment option a reality for the AHP community. We also commend the FDA for\nrecognizing the immense medical need and granting this approval so quickly,\u201d\nsaid John Maraganore, Ph.D., Chief Executive Officer of Alnylam. \u201cGIVLAARI now\nbecomes our second RNAi therapeutic to be approved in the last 16 months, and\nthe world\u2019s first-ever GalNAc-conjugate RNA therapeutic to be approved,\nrepresenting a watershed moment for a technology uniquely pioneered by Alnylam\nscientists. We believe today\u2019s news reinforces the promise and potential of\nRNAi therapeutics as a whole new class of medicines and brings us one important\nstep closer to fulfilling our&nbsp;<em>Alnylam 2020<\/em>&nbsp;goals of building a\nmulti-product, global commercial company with a deep clinical pipeline to drive\ngrowth and an organic product engine to fuel sustainable innovation.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The FDA approval of\nGIVLAARI was received in less than four months after acceptance of the NDA, and\nwas based on positive results from the ENVISION Phase 3 study, a randomized,\ndouble-blind, placebo-controlled, multinational study of 94 patients with AHP,\nat 36 study sites in 18 countries \u2013 the largest ever interventional study\nconducted in AHP. In ENVISION, AHP patients on GIVLAARI experienced 70% (95%\nCI: 60%, 80%) fewer porphyria attacks compared to placebo. GIVLAARI also\nresulted in a similar reduction in intravenous hemin use, as well as reductions\nin urinary aminolevulinic acid (ALA), and urinary porphobilinogen (PBG).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In the pivotal\nENVISION study, the most common adverse reactions (reported in at least 20% of\npatients) with GIVLAARI were nausea (27%) and injection site reactions (25%).\nOther adverse reactions seen in patients treated with GIVLAARI (occurring over\n5% more frequently than placebo) include rash, serum creatinine increase,\ntransaminase elevations and fatigue. As previously reported, one patient in the\nGIVLAARI clinical development program experienced an anaphylactic reaction\nwhich resolved with medical management.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cAdults with AHP now\nhave a new treatment option that has demonstrated the ability to reduce the\nfrequency of porphyria attacks by specifically addressing factors associated\nwith attacks and other disease manifestations of AHP,\u201d said Manisha Balwani<sup>i<\/sup>, M.D., M.S, Associate Professor of the Department of Genetics\nand Genomic Sciences and Department of Medicine at the Icahn School of Medicine\nat Mount Sinai and principal investigator of the ENVISION study. \u201cWith the approval\nof GIVLAARI, and based on the efficacy data from the ENVISION study, I hope to\nsee my patients and those across the country be able to live more normal lives\nwith fewer porphyria attacks.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThe FDA approval of\nGIVLAARI is an important milestone for our community, as we now have a new\ntreatment option for adults living with acute hepatic porphyria,\u201d said Kristen\nWheeden, Executive Director, American Porphyria Foundation. \u201cAHP can have a\nprofound impact on the lives of patients and their families. Porphyria attacks\nare associated with severe, incapacitating pain, often requiring\nhospitalization for management. In addition, many patients struggle on a daily\nbasis with chronic symptoms related to their disease. The approval of GIVLAARI\nis exciting for our community.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Alnylam is committed\nto helping people access the medicines they are prescribed and will be offering\ncomprehensive support services for people prescribed GIVLAARI through Alnylam\nAssist<sup>\u00ae<\/sup>. Visit AlnylamAssist.com for more information\nor call 1-833-256-2748.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">GIVLAARI is expected\nto be available for shipment to healthcare providers in the U.S. by year-end.\nHCPs can initiate the process now by visiting&nbsp;<a href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=http%3A%2F%2Fwww.AlnylamAssist.com&amp;esheet=52125392&amp;newsitemid=20191120005849&amp;lan=en-US&amp;anchor=www.AlnylamAssist.com&amp;index=2&amp;md5=25fc4a2ef7feb792028ad9716453f553\" target=\"_blank\" rel=\"noreferrer noopener\">www.AlnylamAssist.com<\/a>&nbsp;and\ncompleting and submitting a Start Form.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In August,\nAlnylam&nbsp;<a href=\"https:\/\/cts.businesswire.com\/ct\/CT?id=smartlink&amp;url=http%3A%2F%2Finvestors.alnylam.com%2Fnews-releases%2Fnews-release-details%2Falnylam-pharmaceuticals-and-ironwood-pharmaceuticals-enter-us-gi&amp;esheet=52125392&amp;newsitemid=20191120005849&amp;lan=en-US&amp;anchor=announced&amp;index=3&amp;md5=678a1f5c7d1f4bd4ab73d5710e57aa9a\" target=\"_blank\" rel=\"noreferrer noopener\">announced<\/a>&nbsp;a U.S.\ngastrointestinal (GI) disease education and promotional agreement for GIVLAARI\nwith Ironwood Pharmaceuticals, Inc., a GI healthcare company. Under the\nagreement, Alnylam will leverage Ironwood\u2019s leading capabilities in GI to help\nraise awareness of AHP among gastroenterologists and other healthcare\npractitioners in the U.S. Ironwood will also participate in GIVLAARI\npromotional efforts, augmenting Alnylam\u2019s broader commercialization activities.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">GIVLAARI was reviewed by the FDA under Priority Review and had previously been granted Breakthrough Therapy and Orphan Drug Designations in the U.S. GIVLAARI is currently being reviewed under accelerated assessment by the European Medicines Agency (EMA) for the treatment of patients with AHP, after receiving Priority Medicines (PRIME) Designation and Orphan Drug Designation from the EMA.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Source: Alnylam (press release)<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Alnylam has received FDA approval for GIVLAARI\u2122&nbsp;(givosiran) for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. Long-term complications of AHP can include chronic neuropathic pain, [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[39],"tags":[],"class_list":["post-16124","post","type-post","status-publish","format-standard","hentry","category-gezondheid"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Alnylam\u2019s GIVLAARI receives FDA approval - hollandbio<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Alnylam\u2019s GIVLAARI receives FDA approval - hollandbio\" \/>\n<meta property=\"og:description\" content=\"Alnylam has received FDA approval for GIVLAARI\u2122&nbsp;(givosiran) for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. Long-term complications of AHP can include chronic neuropathic pain, [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/\" \/>\n<meta property=\"og:site_name\" content=\"hollandbio\" \/>\n<meta property=\"article:published_time\" content=\"2019-11-26T21:43:51+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.hollandbio.nl\/wp-content\/uploads\/2025\/01\/hb-opengraph_illu.png\" \/>\n\t<meta property=\"og:image:width\" content=\"1200\" \/>\n\t<meta property=\"og:image:height\" content=\"675\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"hollandbio\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@hollandbio\" \/>\n<meta name=\"twitter:site\" content=\"@hollandbio\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"hollandbio\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"4 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/\"},\"author\":{\"name\":\"hollandbio\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#\\\/schema\\\/person\\\/833d6a25b39946e61c6101af1fa96fbb\"},\"headline\":\"Alnylam\u2019s GIVLAARI receives FDA approval\",\"datePublished\":\"2019-11-26T21:43:51+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/\"},\"wordCount\":845,\"publisher\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#organization\"},\"articleSection\":[\"Gezondheid\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/\",\"url\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/\",\"name\":\"Alnylam\u2019s GIVLAARI receives FDA approval - hollandbio\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#website\"},\"datePublished\":\"2019-11-26T21:43:51+00:00\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/nieuws\\\/alnylams-givlaari-receives-fda-approval\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Alnylam\u2019s GIVLAARI receives FDA approval\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#website\",\"url\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/\",\"name\":\"hollandbio\",\"description\":\"\",\"publisher\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"},{\"@type\":\"Organization\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#organization\",\"name\":\"hollandbio\",\"url\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#\\\/schema\\\/logo\\\/image\\\/\",\"url\":\"https:\\\/\\\/www.hollandbio.nl\\\/wp-content\\\/uploads\\\/2023\\\/08\\\/HollandBIO_logo.svg\",\"contentUrl\":\"https:\\\/\\\/www.hollandbio.nl\\\/wp-content\\\/uploads\\\/2023\\\/08\\\/HollandBIO_logo.svg\",\"width\":218.664,\"height\":29.659,\"caption\":\"hollandbio\"},\"image\":{\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#\\\/schema\\\/logo\\\/image\\\/\"},\"sameAs\":[\"https:\\\/\\\/x.com\\\/hollandbio\",\"https:\\\/\\\/www.linkedin.com\\\/company\\\/hollandbio\\\/\"]},{\"@type\":\"Person\",\"@id\":\"https:\\\/\\\/www.hollandbio.nl\\\/en\\\/#\\\/schema\\\/person\\\/833d6a25b39946e61c6101af1fa96fbb\",\"name\":\"hollandbio\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Alnylam\u2019s GIVLAARI receives FDA approval - hollandbio","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/","og_locale":"en_US","og_type":"article","og_title":"Alnylam\u2019s GIVLAARI receives FDA approval - hollandbio","og_description":"Alnylam has received FDA approval for GIVLAARI\u2122&nbsp;(givosiran) for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. Long-term complications of AHP can include chronic neuropathic pain, [&hellip;]","og_url":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/","og_site_name":"hollandbio","article_published_time":"2019-11-26T21:43:51+00:00","og_image":[{"width":1200,"height":675,"url":"https:\/\/www.hollandbio.nl\/wp-content\/uploads\/2025\/01\/hb-opengraph_illu.png","type":"image\/png"}],"author":"hollandbio","twitter_card":"summary_large_image","twitter_creator":"@hollandbio","twitter_site":"@hollandbio","twitter_misc":{"Written by":"hollandbio","Est. reading time":"4 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/#article","isPartOf":{"@id":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/"},"author":{"name":"hollandbio","@id":"https:\/\/www.hollandbio.nl\/en\/#\/schema\/person\/833d6a25b39946e61c6101af1fa96fbb"},"headline":"Alnylam\u2019s GIVLAARI receives FDA approval","datePublished":"2019-11-26T21:43:51+00:00","mainEntityOfPage":{"@id":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/"},"wordCount":845,"publisher":{"@id":"https:\/\/www.hollandbio.nl\/en\/#organization"},"articleSection":["Gezondheid"],"inLanguage":"en-US"},{"@type":"WebPage","@id":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/","url":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/","name":"Alnylam\u2019s GIVLAARI receives FDA approval - hollandbio","isPartOf":{"@id":"https:\/\/www.hollandbio.nl\/en\/#website"},"datePublished":"2019-11-26T21:43:51+00:00","breadcrumb":{"@id":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/www.hollandbio.nl\/en\/nieuws\/alnylams-givlaari-receives-fda-approval\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/www.hollandbio.nl\/en\/"},{"@type":"ListItem","position":2,"name":"Alnylam\u2019s GIVLAARI receives FDA approval"}]},{"@type":"WebSite","@id":"https:\/\/www.hollandbio.nl\/en\/#website","url":"https:\/\/www.hollandbio.nl\/en\/","name":"hollandbio","description":"","publisher":{"@id":"https:\/\/www.hollandbio.nl\/en\/#organization"},"potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/www.hollandbio.nl\/en\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"},{"@type":"Organization","@id":"https:\/\/www.hollandbio.nl\/en\/#organization","name":"hollandbio","url":"https:\/\/www.hollandbio.nl\/en\/","logo":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/www.hollandbio.nl\/en\/#\/schema\/logo\/image\/","url":"https:\/\/www.hollandbio.nl\/wp-content\/uploads\/2023\/08\/HollandBIO_logo.svg","contentUrl":"https:\/\/www.hollandbio.nl\/wp-content\/uploads\/2023\/08\/HollandBIO_logo.svg","width":218.664,"height":29.659,"caption":"hollandbio"},"image":{"@id":"https:\/\/www.hollandbio.nl\/en\/#\/schema\/logo\/image\/"},"sameAs":["https:\/\/x.com\/hollandbio","https:\/\/www.linkedin.com\/company\/hollandbio\/"]},{"@type":"Person","@id":"https:\/\/www.hollandbio.nl\/en\/#\/schema\/person\/833d6a25b39946e61c6101af1fa96fbb","name":"hollandbio"}]}},"_links":{"self":[{"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/posts\/16124","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/users\/6"}],"replies":[{"embeddable":true,"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/comments?post=16124"}],"version-history":[{"count":0,"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/posts\/16124\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/media?parent=16124"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/categories?post=16124"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.hollandbio.nl\/en\/wp-json\/wp\/v2\/tags?post=16124"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}