{"id":948,"date":"2022-05-02T12:00:00","date_gmt":"2022-05-02T12:00:00","guid":{"rendered":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/?p=948"},"modified":"2026-10-30T06:59:24","modified_gmt":"2026-10-30T06:59:24","slug":"12-2","status":"publish","type":"post","link":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/news\/12-2\/","title":{"rendered":"Orphalan announces FDA approval of Cuvrior\u2122 for the treatment of adult patients with stable Wilson&#8217;s disease who are de-coppered and tolerant to penicillamine"},"content":{"rendered":"<div class=\"od-news-story\" data-source-id=\"12\" data-hcp=\"false\">\n<aside class=\"od-news-summary\">\n<p class=\"od-news-summary-copy\">Orphalan announces FDA approval of Cuvrior\u2122 for the treatment of adult patients with stable Wilson&#8217;s disease who are de-coppered and tolerant to penicillamine.<\/p>\n<\/aside>\n<p class=\"od-news-p\">Paris, France 2 May 2022 \u2013 Orphalan SA (&quot;Orphalan&quot; or &quot;the Company&quot;), an international orphan drug development and commercialisation company, today announces approval of Cuvrior\u2122, a new salt of trientine (trientine tetrahydrochloride) by the United States Food and Drug Administration (FDA). Cuvrior\u2122 is approved for the treatment of adult patients with stable Wilson&#8217;s disease who are de-coppered and tolerant to penicillamine. Penicillamine is currently approved as a first-line treatment of Wilson&#8217;s disease in the US with about one third of patients developing intolerance1.<\/p>\n<p class=\"od-news-p\">Orphalan recently completed a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior\u2122 was non-inferior to penicillamine as measured by non-ceruloplasmin copper (NCC). In consultation with the FDA, an assay based on total serum copper protein speciation was used for measuring this primary efficacy endpoint.<\/p>\n<p class=\"od-news-p\">Wilson&#8217;s disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Orphalan commercializes its trientine tetrahydrochloride product in Europe under the name of Cuprior\u00ae and expects to launch Cuvrior\u2122, which has been granted Orphan Drug Designation by the FDA, in the US by early 2023. The approval follows the New Drug Application (NDA) submission for the company&#8217;s product last year.<\/p>\n<blockquote class=\"od-news-quote\"><p><svg class=\"od-news-quote-icon\" aria-hidden=\"true\" viewbox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M16 3a2 2 0 0 0-2 2v6a2 2 0 0 0 2 2 1 1 0 0 1 1 1v1a2 2 0 0 1-2 2 1 1 0 0 0-1 1v2a1 1 0 0 0 1 1 6 6 0 0 0 6-6V5a2 2 0 0 0-2-2z\"><\/path><path d=\"M5 3a2 2 0 0 0-2 2v6a2 2 0 0 0 2 2 1 1 0 0 1 1 1v1a2 2 0 0 1-2 2 1 1 0 0 0-1 1v2a1 1 0 0 0 1 1 6 6 0 0 0 6-6V5a2 2 0 0 0-2-2z\"><\/path><\/svg><\/p>\n<p class=\"od-news-quote-copy\">&quot;We are delighted with the approval of our product, Cuvrior\u2122, which provides a well-tolerated and effective option for Wilson&#8217;s disease patients. At Orphalan, we are committed to delivering innovative therapies, with our drug Cuprior\u00ae already approved and launched in European countries, and we look forward to launching Cuvrior\u2122 in the United States. We also plan further national submissions to make our product available to patients globally.&quot;<\/p>\n<p><cite class=\"od-news-cite\">&#8211; Dr. Naseem Amin, Chief Executive Officer at Orphalan<\/cite><\/p><\/blockquote>\n<blockquote class=\"od-news-quote\"><p><svg class=\"od-news-quote-icon\" aria-hidden=\"true\" viewbox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M16 3a2 2 0 0 0-2 2v6a2 2 0 0 0 2 2 1 1 0 0 1 1 1v1a2 2 0 0 1-2 2 1 1 0 0 0-1 1v2a1 1 0 0 0 1 1 6 6 0 0 0 6-6V5a2 2 0 0 0-2-2z\"><\/path><path d=\"M5 3a2 2 0 0 0-2 2v6a2 2 0 0 0 2 2 1 1 0 0 1 1 1v1a2 2 0 0 1-2 2 1 1 0 0 0-1 1v2a1 1 0 0 0 1 1 6 6 0 0 0 6-6V5a2 2 0 0 0-2-2z\"><\/path><\/svg><\/p>\n<p class=\"od-news-quote-copy\">&quot;Wilson&#8217;s disease is a devastating disorder affecting patients worldwide and for which there has remained a significant need for innovative new treatments. The approval of Orphalan&#8217;s Cuvrior\u2122 is extremely promising and is reflective of Orphalan&#8217;s patient-driven approach. This marks the culmination of many years of work and is an important moment, offering new hope for patients affected by this disease.&quot;<\/p>\n<p><cite class=\"od-news-cite\">&#8211; Mary L Graper, Vice President of Scientific Affairs, Wilson Disease Association<\/cite><\/p><\/blockquote>\n<blockquote class=\"od-news-quote\"><p><svg class=\"od-news-quote-icon\" aria-hidden=\"true\" viewbox=\"0 0 24 24\" fill=\"none\" stroke=\"currentColor\" stroke-width=\"2\" stroke-linecap=\"round\" stroke-linejoin=\"round\"><path d=\"M16 3a2 2 0 0 0-2 2v6a2 2 0 0 0 2 2 1 1 0 0 1 1 1v1a2 2 0 0 1-2 2 1 1 0 0 0-1 1v2a1 1 0 0 0 1 1 6 6 0 0 0 6-6V5a2 2 0 0 0-2-2z\"><\/path><path d=\"M5 3a2 2 0 0 0-2 2v6a2 2 0 0 0 2 2 1 1 0 0 1 1 1v1a2 2 0 0 1-2 2 1 1 0 0 0-1 1v2a1 1 0 0 0 1 1 6 6 0 0 0 6-6V5a2 2 0 0 0-2-2z\"><\/path><\/svg><\/p>\n<p class=\"od-news-quote-copy\">&quot;As a physician, I have seen first-hand how Wilson&#8217;s disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available. The approval of Orphalan&#8217;s Cuvrior\u2122 by the FDA is backed by positive data from Orphalan&#8217;s multicenter, multinational CHELATE trial \u2013 the first head-to-head controlled study of a new trientine salt versus penicillamine. For patients in need, Cuvrior\u2122 represents a well-tolerated and effective alternative to penicillamine, the current standard of care.&quot;<\/p>\n<p><cite class=\"od-news-cite\">&#8211; Professor Michael Schilsky, MD, Director, Center for Excellence for Wilson Disease at Yale University<\/cite><\/p><\/blockquote>\n<aside class=\"od-news-note\">\n<p class=\"od-news-note-copy\">1 Weiss KH, et al. Efficacy and Safety of Oral Chelators in Treatment of Patients With Wilson Disease. Clin Gastroenterol Hepatol. 2013 Aug;11(8):1028-35.e1-2.<\/p>\n<\/aside>\n<h2 class=\"od-news-heading\">About Trientine Tetrahydrochloride<\/h2>\n<p class=\"od-news-p\">Trientine tetrahydrochloride is an oral trientine formulation. In the US, trientine tetrahydrochloride has been granted with Orphan Drug Designation for the treatment of Wilson&#8217;s disease excluding patients intolerant of penicillamine. It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilson&#8217;s disease patients who are successfully de-coppered and tolerant to penicillamine. The 505(b)(2) regulatory pathway is a type of New Drug Application (NDA).<\/p>\n<h2 class=\"od-news-heading\">About Orphalan<\/h2>\n<p class=\"od-news-p\">Orphalan is an international orphan drug development and commercialisation company. The company delivers worldwide innovative therapies for people living with orphan diseases and is a pioneer in the space. Orphalan was founded in 2011 and has launched Cuprior\u00ae across Europe with its own commercial organization. For more information visit www.orphalan.com and follow us on LinkedIn.<\/p>\n<aside class=\"od-news-contact\">\n<p class=\"od-news-contact-copy\">For more information, please contact:<br \/>\nOrphalan<br \/>\nTel: +33 (0)1 42 49 82 64<br \/>\ninfo@orphalan.com<\/p>\n<p>Consilium Strategic Communications:<br \/>\nMary-Jane Elliott, Davide Salvi, Genevieve Wilson<br \/>\nTel: +44 (0) 203 709 5700<br \/>\norphalan@consilium-comms.com<\/p>\n<\/aside>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Orphalan announces FDA approval of Cuvrior\u2122 for the treatment of adult patients with stable Wilson&#8217;s disease who are de-coppered and tolerant to penicillamine.<\/p>","protected":false},"author":1,"featured_media":95,"comment_status":"closed","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"_oxygen_hide_in_design_set":false,"_oxygen_tags":"","footnotes":""},"categories":[3],"tags":[],"class_list":["post-948","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-release"],"_links":{"self":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/posts\/948","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/comments?post=948"}],"version-history":[{"count":1,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/posts\/948\/revisions"}],"predecessor-version":[{"id":949,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/posts\/948\/revisions\/949"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/media\/95"}],"wp:attachment":[{"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/media?parent=948"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/categories?post=948"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/funny-fermi.104-248-174-66.plesk.page\/fr\/wp-json\/wp\/v2\/tags?post=948"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}