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Overview | Eplontersen for treating hereditary transthyretin-related amyloidosis | Guidance | NICE
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<div id="toTop"><span aria-hidden="true" class="icon-nice-chevron-up"></span></div> <div class="_"> <div class="content"> <div class="zone zone-content"> <div class="container"> <nav class="horizontal-nav hide-print" aria-label="Sections within this product"> <ul class="horizontal-nav__list"> <li class="horizontal-nav__item"> <a aria-current="true" class="horizontal-nav__link" href="/guidance/ta1020">Guidance</a> </li> <li class="horizontal-nav__item"> <a aria-current="false" class="horizontal-nav__link" href="/guidance/ta1020/resources">Tools and resources</a> </li> <li class="horizontal-nav__item"> <a aria-current="false" class="horizontal-nav__link" href="/guidance/ta1020/informationforpublic">Information for the public</a> </li> <li class="horizontal-nav__item"> <a aria-current="false" class="horizontal-nav__link" href="/guidance/ta1020/evidence">Evidence</a> </li> <li class="horizontal-nav__item"> <a aria-current="false" class="horizontal-nav__link" href="/guidance/ta1020/history">History</a> </li> </ul> </nav> <div class="grid grid--loose"> <div data-g="12 sm:5 md:3" class="hide-print"> <a href="/guidance/ta1020/resources/eplontersen-for-treating-hereditary-transthyretinrelated-amyloidosis-pdf-2973528327509701" target="_blank" class="btn btn--cta mr--0 mb--e mb--d-sm show-ib show-sm text-center" data-track="guidancedownload" data-track-category="guidance download" data-track-action="clicked" data-track-label="/guidance/ta1020/resources/eplontersen-for-treating-hereditary-transthyretinrelated-amyloidosis-pdf-2973528327509701"> Download guidance (PDF) </a> <nav class="stacked-nav" aria-label="Chapters"> <ul class="stacked-nav__list"> <li class="stacked-nav__list-item"> <a aria-current="true" href="/guidance/ta1020"> <span class="stacked-nav__content-wrapper"> Overview </span> </a> </li> <li class="stacked-nav__list-item"> <a aria-current="false" href="/guidance/ta1020/chapter/1-Recommendations"> <span class="stacked-nav__content-wrapper">1 Recommendations</span> </a> </li> <li class="stacked-nav__list-item"> <a aria-current="false" href="/guidance/ta1020/chapter/2-Information-about-eplontersen"> <span class="stacked-nav__content-wrapper">2 Information about eplontersen</span> </a> </li> <li class="stacked-nav__list-item"> <a aria-current="false" href="/guidance/ta1020/chapter/3-Implementation"> <span class="stacked-nav__content-wrapper">3 Implementation</span> </a> </li> <li class="stacked-nav__list-item"> <a aria-current="false" href="/guidance/ta1020/chapter/4-Evaluation-committee-members-and-NICE-project-team"> <span class="stacked-nav__content-wrapper">4 Evaluation committee members and NICE project team</span> </a> </li> </ul> </nav> </div> <div data-g="12 sm:7 md:9"> <div class="grid grid--loose"> <div data-g="12 md:8"> <div class="grid grid--loose grid--rev"> <div data-g="12"> <h2 class="mt--0">Overview</h2> <div class="mb--e js-in-page-nav-target"> <p class="lead"><a href="https://www.nice.org.uk/guidance/TA1020/chapter/1-Recommendations">Evidence-based recommendations</a> on eplontersen (Wainzua) for treating hereditary transthyretin-related amyloidosis in adults.</p> <p><strong>Last reviewed:</strong> 27 November 2024</p> <p><strong>Next review: </strong>This guidance will be reviewed if there is new evidence that is likely to change the recommendations.</p> <h3>Commercial arrangement</h3> <p>There is a simple discount patient access scheme for eplontersen. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact <a href="mailto:Market.AccessUK@astrazeneca.com">Market.AccessUK@astrazeneca.com</a> for details.</p> <h3>Guidance development process</h3> <p><a href="https://www.nice.org.uk/About/What-we-do/Our-Programmes/NICE-guidance/NICE-technology-appraisal-guidance">How we develop NICE technology appraisal guidance</a></p><div class='panel'><h4 class="title">Your responsibility</h4><p>The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance is at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.</p><p>All problems (adverse events) related to a medicine or medical device used for treatment or in a procedure should be reported to the Medicines and Healthcare products Regulatory Agency using the <a href="https://www.gov.uk/report-problem-medicine-medical-device">Yellow Card Scheme</a>.</p><p>Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities.</p><p>Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should <a href="https://www.nice.org.uk/about/who-we-are/sustainability">assess and reduce the environmental impact of implementing NICE recommendations</a> wherever possible.</p></div> </div> </div> </div> </div> <div data-g="12" class="hide-print"> <hr class="mt--c" /> <div class="prev-next"> <nav aria-label="Previous and next pages"> <a class="prev-next__link" href="/guidance/ta1020/chapter/1-Recommendations" rel="next"> <span class="prev-next__link-intro">Next page</span> <span class="prev-next__link-text">1 Recommendations</span> </a> </nav> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> </main> <div id="global-nav-footer"></div> <script> var global_nav_config = { service: "guidance", header: { cookie: false, skipLinkId: "content-start", search: { url: "/search", autocomplete: "/autocomplete?ajax=ajax", }, auth: { environment: "live" } } }; </script> <!--[if lt IE 9]> <script src="https://cdn.nice.org.uk/global-nav/global-nav.ie8.min.js" type="text/javascript"></script> <![endif]--> <script src="https://cdn.nice.org.uk/global-nav/global-nav.min.js" type="text/javascript"></script> <script src="//cdn.nice.org.uk/V2/Scripts/twitter.bootstrap.min.js" type="text/javascript"></script> <script src="/Themes/NICE.Bootstrap/scripts/niceorg/NICE.TopScroll.js" type="text/javascript"></script> <script src="//cdn.nice.org.uk/V2/Scripts/NICE.bootstrap.min.js" type="text/javascript"></script> <script src="https://cdn.nice.org.uk/niceorg/js/app.js" async></script> </body> </html>