{"id":541137,"date":"2021-09-22T11:20:04","date_gmt":"2021-09-22T15:20:04","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/vertexs-supplement-to-a-new-drug-submission-for-kalydeco-ivacaftor-for-patients-with-cystic-fibrosis-between-the-ages-of-4-months-and-18-years-with-the-r117h-mutation-in-the-cftr-gene-accept\/"},"modified":"2021-09-22T11:20:04","modified_gmt":"2021-09-22T15:20:04","slug":"vertexs-supplement-to-a-new-drug-submission-for-kalydeco-ivacaftor-for-patients-with-cystic-fibrosis-between-the-ages-of-4-months-and-18-years-with-the-r117h-mutation-in-the-cftr-gene-accept","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/vertexs-supplement-to-a-new-drug-submission-for-kalydeco-ivacaftor-for-patients-with-cystic-fibrosis-between-the-ages-of-4-months-and-18-years-with-the-r117h-mutation-in-the-cftr-gene-accept\/","title":{"rendered":"Vertex&#8217;s Supplement to a New Drug Submission for KALYDECO\u00ae (ivacaftor) for Patients with Cystic Fibrosis Between the Ages of 4 Months and 18 Years with the R117H Mutation in the CFTR Gene Accepted for Priority Review by Health Canada"},"content":{"rendered":"<div class=\"xn-newslines\">\n<p class=\"xn-distributor\">Canada NewsWire<\/p>\n<\/p><\/div>\n<div class=\"xn-content\">\n<p>\n        <span class=\"xn-location\">TORONTO<\/span>, <span class=\"xn-chron\">Sept. 22, 2021<\/span> \/CNW\/ &#8211; <u><a target=\"_blank\" href=\"https:\/\/c212.net\/c\/link\/?t=0&amp;l=en&amp;o=3299368-1&amp;h=1592094845&amp;u=https%3A%2F%2Fwww.vrtx.com%2F&amp;a=Vertex+Pharmaceuticals+Incorporated%C2%A0(Canada)\" rel=\"nofollow noopener\">Vertex Pharmaceuticals Incorporated\u00a0(<span class=\"xn-location\">Canada<\/span>)<\/a><\/u>\u00a0(Nasdaq: VRTX) today announced its Supplement to a New Drug Submission for <sup>Pr<\/sup>KALYDECO<sup>\u00ae<\/sup> (ivacaftor) has been accepted for priority review by Health Canada for the treatment of cystic fibrosis (CF) in patients from 4 months to 18 years of age and weighing at least 5 kg with the <i>R117H<\/i> mutation in the cystic fibrosis transmembrane conductance regulator (<i>CFTR<\/i>) gene.<\/p>\n<p>&#8220;We are pleased this submission has been accepted for Priority Review by Health Canada, and we hope that this will enable access for patients with the R117H mutation as soon as possible,&#8221; said <span class=\"xn-person\">Duncan McKechnie<\/span>, Senior Vice President, North America Commercial Operations, Vertex Pharmaceuticals.\u00a0&#8220;It&#8217;s our goal to ensure as many people as possible with cystic fibrosis are able to access treatments for the underlying cause of their CF.&#8221;<\/p>\n<p>\n        <b>About Cystic Fibrosis<\/b>\n      <\/p>\n<p>Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and\/or missing CFTR protein resulting from certain mutations in the\u00a0<i>CFTR<\/i>\u00a0gene. Children must inherit two defective\u00a0<i>CFTR<\/i>\u00a0genes \u2014 one from each parent \u2014 to have CF. While there are many different types of\u00a0<i>CFTR<\/i>\u00a0mutations that can cause the disease, the vast majority of all people with CF have at least one\u00a0<i>F508del<\/i>\u00a0mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and\/or too few CFTR proteins at the cell surface. The defective function and\/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.<\/p>\n<p>\n        <b>About KALYDECO<sup>\u00ae<\/sup> (ivacaftor)<\/b>\n      <\/p>\n<p>Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the\u00a0<i>CFTR<\/i>\u00a0gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.<\/p>\n<p>\n        <b>About\u00a0Vertex<\/b>\n      <\/p>\n<p>Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) \u2014 a rare, life-threatening genetic disease \u2014 and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.<\/p>\n<p>Founded in 1989 in\u00a0Cambridge, Mass., Vertex&#8217;s global headquarters is now located in\u00a0Boston&#8217;s\u00a0Innovation District and its international headquarters is in\u00a0London. Additionally, the company has research and development sites and commercial offices in\u00a0North America,\u00a0Europe,\u00a0Australia\u00a0and\u00a0Latin America. Vertex is consistently recognized as one of the industry&#8217;s top places to work, including 11 consecutive years on Science magazine&#8217;s Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. <\/p>\n<p>\n        <b>Special Note Regarding Forward-Looking Statements<\/b>\n      <\/p>\n<p>This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by <span class=\"xn-person\">Duncan McKechnie<\/span> in this press release, including expectations for patient access to our medicine. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company&#8217;s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the New Drug Submission to Health Canada may not be approved in the expected timeline, or at all, that data from the company&#8217;s development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading &#8220;Risk Factors&#8221; in Vertex&#8217;s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at <u><a target=\"_blank\" href=\"https:\/\/c212.net\/c\/link\/?t=0&amp;l=en&amp;o=3299368-1&amp;h=3816403076&amp;u=https%3A%2F%2Fwww.sec.gov%2F&amp;a=www.sec.gov\" rel=\"nofollow noopener\">www.sec.gov<\/a><\/u> and available through the company&#8217;s website at <u><a target=\"_blank\" href=\"https:\/\/c212.net\/c\/link\/?t=0&amp;l=en&amp;o=3299368-1&amp;h=824530258&amp;u=http%3A%2F%2Fwww.vrtx.com%2F&amp;a=www.vrtx.com\" rel=\"nofollow noopener\">www.vrtx.com<\/a><\/u>. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. <\/p>\n<p>(VRTX-GEN)<\/p>\n<p>SOURCE  Vertex Pharmaceuticals Incorporated (<span class=\"xn-location\">Canada<\/span>)<\/p>\n<\/p><\/div>\n<p>    <img decoding=\"async\" alt=\"\" src=\"https:\/\/rt.prnewswire.com\/rt.gif?NewsItemId=C0015&amp;Transmission_Id=202109221116CANADANWWEB______C0015&amp;DateId=20210922\" style=\"border:0px;width:1px;height:1px\" \/><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Canada NewsWire TORONTO, Sept. 22, 2021 \/CNW\/ &#8211; Vertex Pharmaceuticals Incorporated\u00a0(Canada)\u00a0(Nasdaq: VRTX) today announced its Supplement to a New Drug Submission for PrKALYDECO\u00ae (ivacaftor) has been accepted for priority review by Health Canada for the treatment of cystic fibrosis (CF) in patients from 4 months to 18 years of age and weighing at least 5 kg with the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. &#8220;We are pleased this submission has been accepted for Priority Review by Health Canada, and we hope that this will enable access for patients with the R117H mutation as soon as possible,&#8221; said Duncan McKechnie, Senior Vice President, North America Commercial Operations, Vertex Pharmaceuticals.\u00a0&#8220;It&#8217;s our goal to ensure as many people &hellip; <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/www.marketnewsdesk.com\/index.php\/vertexs-supplement-to-a-new-drug-submission-for-kalydeco-ivacaftor-for-patients-with-cystic-fibrosis-between-the-ages-of-4-months-and-18-years-with-the-r117h-mutation-in-the-cftr-gene-accept\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> &#8220;Vertex&#8217;s Supplement to a New Drug Submission for KALYDECO\u00ae (ivacaftor) for Patients with Cystic Fibrosis Between the Ages of 4 Months and 18 Years with the R117H Mutation in the CFTR Gene Accepted for Priority Review by Health Canada&#8221;<\/span><\/a><\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[],"tags":[],"class_list":["post-541137","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Vertex&#039;s Supplement to a New Drug Submission for KALYDECO\u00ae (ivacaftor) for Patients with Cystic Fibrosis Between the Ages of 4 Months and 18 Years with the R117H Mutation in the CFTR Gene Accepted for Priority Review by Health Canada - Market Newsdesk<\/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.marketnewsdesk.com\/index.php\/vertexs-supplement-to-a-new-drug-submission-for-kalydeco-ivacaftor-for-patients-with-cystic-fibrosis-between-the-ages-of-4-months-and-18-years-with-the-r117h-mutation-in-the-cftr-gene-accept\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Vertex&#039;s Supplement to a New Drug Submission for KALYDECO\u00ae (ivacaftor) for Patients with Cystic Fibrosis Between the Ages of 4 Months and 18 Years with the R117H Mutation in the CFTR Gene Accepted for Priority Review by Health Canada - Market Newsdesk\" \/>\n<meta property=\"og:description\" content=\"Canada NewsWire TORONTO, Sept. 22, 2021 \/CNW\/ &#8211; Vertex Pharmaceuticals Incorporated\u00a0(Canada)\u00a0(Nasdaq: VRTX) today announced its Supplement to a New Drug Submission for PrKALYDECO\u00ae (ivacaftor) has been accepted for priority review by Health Canada for the treatment of cystic fibrosis (CF) in patients from 4 months to 18 years of age and weighing at least 5 kg with the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. &#8220;We are pleased this submission has been accepted for Priority Review by Health Canada, and we hope that this will enable access for patients with the R117H mutation as soon as possible,&#8221; said Duncan McKechnie, Senior Vice President, North America Commercial Operations, Vertex Pharmaceuticals.\u00a0&#8220;It&#8217;s our goal to ensure as many people &hellip; Continue reading &quot;Vertex&#8217;s Supplement to a New Drug Submission for KALYDECO\u00ae (ivacaftor) for Patients with Cystic Fibrosis Between the Ages of 4 Months and 18 Years with the R117H Mutation in the CFTR Gene Accepted for Priority Review by Health Canada&quot;\" \/>\n<meta property=\"og:url\" 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Review by Health Canada - Market Newsdesk","og_description":"Canada NewsWire TORONTO, Sept. 22, 2021 \/CNW\/ &#8211; Vertex Pharmaceuticals Incorporated\u00a0(Canada)\u00a0(Nasdaq: VRTX) today announced its Supplement to a New Drug Submission for PrKALYDECO\u00ae (ivacaftor) has been accepted for priority review by Health Canada for the treatment of cystic fibrosis (CF) in patients from 4 months to 18 years of age and weighing at least 5 kg with the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. &#8220;We are pleased this submission has been accepted for Priority Review by Health Canada, and we hope that this will enable access for patients with the R117H mutation as soon as possible,&#8221; said Duncan McKechnie, Senior Vice President, North America Commercial Operations, Vertex Pharmaceuticals.\u00a0&#8220;It&#8217;s our goal to ensure as many people &hellip; Continue reading \"Vertex&#8217;s Supplement to a New Drug Submission for 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