{"id":449901,"date":"2017-06-19T12:00:00","date_gmt":"2017-06-19T12:00:00","guid":{"rendered":"https:\/\/www.pm360online.com\/?guid=6c76d7f03c338650b63ae9491b57ff7a"},"modified":"2017-06-19T12:00:00","modified_gmt":"2017-06-19T12:00:00","slug":"horizon-pharma-plc-announces-health-canada-approval-of-procysbi-cysteamine-delayed-release-capsules-for-the-treatment-of-nephropathic-cystinosis","status":"publish","type":"post","link":"https:\/\/www.pm360online.com\/horizon-pharma-plc-announces-health-canada-approval-of-procysbi-cysteamine-delayed-release-capsules-for-the-treatment-of-nephropathic-cystinosis\/","title":{"rendered":"Horizon Pharma plc Announces Health Canada Approval of PROCYSBI\u2122\u00a0(Cysteamine Delayed-Release Capsules)\u00a0for the Treatment of Nephropathic Cystinosis"},"content":{"rendered":"

DUBLIN, Ireland, June 19, 2017 (GLOBE NEWSWIRE) — Horizon Pharma plc (NASDAQ:HZNP<\/a>), a biopharmaceutical company focused on improving patients\u2019 lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, and its affiliate\u00a0Horizon Therapeutics Canada, today announced that Health Canada has issued a Notice of Compliance (NOC) for PROCYSBI\u2122\u00a0(cysteamine delayed-release capsules) for the treatment of nephropathic cystinosis in adults and children 2 years of age and older.\u00a0<\/p>\n

\u201cFor children diagnosed with nephropathic cystinosis, treatment with cystine-depleting therapy should be started as soon as possible to avoid the serious and potentially life-threatening impact on tissues and organs,\u201d said Durhane Wong-Rieger, president and chief executive officer, Canadian Organization for Rare Disorders (CORD).\u00a0 \u201cPrior to PROCYSBI there was no approved therapy in Canada for treating cystinosis.\u00a0 We are especially pleased that Health Canada has granted very timely approval of PROCYSBI, the delayed-release form of the therapy, which will help assure patients avoid toxic build-up of cystine in the cells.\u201d
\u00a0\u00a0
PROCYSBI is the only cystine-depleting agent approved in Canada for the treatment\u00a0of nephropathic cystinosis.\u00a0 It is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic concentration of cystine in the cells to limit or prevent the damage too much cystine can cause to cells, tissue and organs.\u00a0<\/p>\n

\u201cThe Cystinosis Research Foundation is proud to have funded every bench and early clinical trial that led to the discovery of PROCYSBI,\u201d said Nancy Stack, chair of the board, Cystinosis Research Foundation.\u00a0 \u201cThe approval of PROCYSBI in Canada is a milestone achievement for the Canadian cystinosis community.\u201d\u00a0<\/p>\n

Cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.\u00a0 Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.\u00a0 It is estimated that only about 2,000 people worldwide are currently diagnosed with cystinosis.\u00a0 Nephropathic or \u201cclassic infantile\u201d cystinosis is the most common and most severe form of the disease, and is typically diagnosed in infancy and requires lifelong therapy.1<\/sup><\/p>\n

\u201cThe Health Canada approval would not have been possible without the dedication of the entire nephropathic cystinosis community, particularly the people who participated in the PROCYSBI clinical studies, their families, as well as advocacy organizations and healthcare professionals,\u201d said\u00a0Jared Rhines, group vice president and general manager,\u00a0Canada, LATAM and APAC, Horizon Pharma plc.\u00a0 \u201cWe look forward to making PROCYSBI available to Canadians living with this extremely challenging disease.\u201d<\/p>\n

About Horizon Pharma plc<\/strong>
Horizon Pharma plc is a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs. \u00a0The Company markets 11 medicines through its orphan, rheumatology and primary care business units. \u00a0For more information, please visit\u00a0www.horizonpharma.com<\/a>. Follow\u00a0@HZNPplc<\/a>\u00a0on Twitter or view careers on our\u00a0LinkedIn<\/a>\u00a0page.<\/p>\n

Forward Looking Statement<\/strong>
This press release contains forward-looking statements, including statements regarding the future availability of PROCYSBI in Canada and the potential of PROCYSBI to treat patients with nephropathic cystinosis.\u00a0 These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors.\u00a0 These factors include whether PROCYSBI will be successfully commercialized and sufficiently available in Canada, as well as those factors described in Horizon Pharma’s filings with the\u00a0United States Securities and Exchange Commission, including those factors discussed under the caption “Risk Factors” in those filings.\u00a0 Forward-looking statements speak only as of the date of this press release and Horizon Pharma does not undertake any obligation to update or revise these statements, except as may be required by law.<\/p>\n

References:<\/u><\/strong><\/p>\n

    \n
  1. Cystinosis Research Foundation. \u201cAbout cystinosis.\u201d \u00a0Available at\u00a0http:\/\/www.cystinosisresearch.org\/About-Cystinosis\/<\/a>. \u00a0Accessed June 16, 2017.<\/li>\n<\/ol>\n
    CONTACT: Contacts:\nTina Ventura\nSenior Vice President, Investor Relations\nInvestor-relations@horizonpharma.com\n\nRuth Venning\nExecutive Director, Investor Relations\nInvestor-relations@horizonpharma.com\n\nU.S. Media Contact:\nMatt Flesch\nExecutive Director, Product Communications\nmedia@horizonpharma.com\n\nIreland Media Contact:\nRay Gordon\nGordon MRM\u00a0\nray@gordonmrm.ie<\/pre>\n","protected":false},"excerpt":{"rendered":"
    DUBLIN, Ireland, June  19, 2017  (GLOBE NEWSWIRE) — Horizon Pharma plc (NASDAQ:HZNP<\/a>), a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, and its affiliate Horizon Therapeutics Canada, today announced that Health Canada has issued a Notice of Compliance (NOC) for PROCYSBI™ (cysteamine delayed-release capsules) for the treatment of nephropathic cystinosis in adults and children 2 years of age and older. <\/p>\n
    “For children diagnosed with nephropathic cystinosis, treatment with cystine-depleting therapy should be started as soon as possible to avoid the serious and potentially life-threatening impact on tissues and organs,” said Durhane Wong-Rieger, president and chief executive officer, Canadian Organization for Rare Disorders (CORD).  “Prior to PROCYSBI there was no approved therapy in Canada for treating cystinosis.  We are especially pleased that Health Canada has granted very timely approval of PROCYSBI, the delayed-release form of the therapy, which will help assure patients avoid toxic build-up of cystine in the cells.”
      
    PROCYSBI is the only cystine-depleting agent approved in Canada for the treatment of nephropathic cystinosis.  It is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic concentration of cystine in the cells to limit or prevent the damage too much cystine can cause to cells, tissue and organs. <\/p>\n
    “The Cystinosis Research Foundation is proud to have funded every bench and early clinical trial that led to the discovery of PROCYSBI,” said Nancy Stack, chair of the board, Cystinosis Research Foundation.  “The approval of PROCYSBI in Canada is a milestone achievement for the Canadian cystinosis community.” <\/p>\n
    Cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.  Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.  It is estimated that only about 2,000 people worldwide are currently diagnosed with cystinosis.  Nephropathic or “classic infantile” cystinosis is the most common and most severe form of the disease, and is typically diagnosed in infancy and requires lifelong therapy.1<\/sup><\/p>\n
    “The Health Canada approval would not have been possible without the dedication of the entire nephropathic cystinosis community, particularly the people who participated in the PROCYSBI clinical studies, their families, as well as advocacy organizations and healthcare professionals,” said Jared Rhines, group vice president and general manager, Canada, LATAM and APAC, Horizon Pharma plc.  “We look forward to making PROCYSBI available to Canadians living with this extremely challenging disease.”<\/p>\n
    About Horizon Pharma plc<\/strong>
    Horizon Pharma plc is a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs.  The Company markets 11 medicines through its orphan, rheumatology and primary care business units.  For more information, please visit     www.horizonpharma.com<\/a>. Follow @HZNPplc<\/a> on Twitter or view careers on our LinkedIn<\/a> page.<\/p>\n
    Forward Looking Statement<\/strong>
    This press release contains forward-looking statements, including statements regarding the future availability of PROCYSBI in Canada and the potential of PROCYSBI to treat patients with nephropathic cystinosis.  These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors.  These factors include whether PROCYSBI will be successfully commercialized and sufficiently available in Canada, as well as those factors described in Horizon Pharma’s filings with the United States Securities and Exchange Commission, including those factors discussed under the caption “Risk Factors” in those filings.  Forward-looking statements speak only as of the date of this press release and Horizon Pharma does not undertake any obligation to update or revise these statements, except as may be required by law.<\/p>\n
    References:<\/u><\/strong><\/p>\n
      \n
    1. Cystinosis Research Foundation. “About cystinosis.”  Available at http:\/\/www.cystinosisresearch.org\/About-Cystinosis\/<\/a>.  Accessed June 16, 2017.<\/li>\n<\/ol>\n
      CONTACT: Contacts:\nTina Ventura\nSenior Vice President, Investor Relations\nInvestor-relations@horizonpharma.com\n\nRuth Venning\nExecutive Director, Investor Relations\nInvestor-relations@horizonpharma.com\n\nU.S. Media Contact:\nMatt Flesch\nExecutive Director, Product Communications\nmedia@horizonpharma.com\n\nIreland Media Contact:\nRay Gordon\nGordon MRM \nray@gordonmrm.ie<\/pre>\n","protected":false},"author":74553,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[71350,71364],"tags":[],"_links":{"self":[{"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/posts\/449901"}],"collection":[{"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/users\/74553"}],"replies":[{"embeddable":true,"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/comments?post=449901"}],"version-history":[{"count":0,"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/posts\/449901\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/media?parent=449901"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/categories?post=449901"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.pm360online.com\/wp-json\/wp\/v2\/tags?post=449901"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}