{"id":20911,"date":"2023-09-04T13:03:48","date_gmt":"2023-09-04T17:03:48","guid":{"rendered":"https:\/\/platohealth.ai\/nice-recommends-new-fabry-disease-treatment\/"},"modified":"2023-09-04T13:56:53","modified_gmt":"2023-09-04T17:56:53","slug":"nice-recommends-new-fabry-disease-treatment","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/nice-recommends-new-fabry-disease-treatment\/","title":{"rendered":"NICE recommends new Fabry disease treatment","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
Recommendation of Elfabrio\u00ae<\/sup> (pegunigalsidase alfa) means patients with Fabry disease in England will be able to access a new treatment option.<\/p>\n<\/div>\n The National Institute for Health and Care Excellence (NICE) has recommended<\/a> Chiesi\u2019s Elfabrio\u00ae<\/sup> (pegunigalsidase alfa) for Fabry disease (alpha-galactosidase deficiency) in adults.<\/p>\n The agreement made in the NICE\u2019s Final Draft Guidance<\/a> means there is now a \u201cnew treatment option for people living with Fabry disease across England,\u201d according to Dr Kamran Iqbal, Head of Medical Affairs for Global Rare Diseases at Chiesi UK&I. \u201cFabry disease brings a multitude of complex symptoms and, since one therapy may not suit all, it is vital that patients have additional treatment options available to them.\u201d<\/p>\n Pegunigalsidase alfa is a novel enzyme replacement therapy (ERT) administered via intravenous infusion every two weeks and delivers a modified version of the enzyme \u03b1-galactosidase A.<\/p>\n Data highlighted by Chiesi noted that the efficacy and safety profile of pegunigalsidase alfa was drawn from the outcomes of a clinical trials programme. Overall, it evaluated 142 patients with Fabry disease (94 males and 48 females). There were 112 patients who received pegunigalsidase alfa 1mg\/kg every other week.<\/p>\n In February 2023, the Committee for Medicinal Products for Human Use (CHMP) recommended<\/a> the first pegylated enzyme for Fabry disease, which is a rare condition. The EMA\u2019s human medicines committee reviewed Phase III clinical data, which combined, represented over 400 years of exposure to PRX\u2013102 (pegunigalsidase alfa).<\/p>\n Several months later, in May, pegunigalsidase alfa was given a marketing authorisation by the European Commission (EC). At the time, Dror Bashan, Protalix\u2019s President and Chief Executive Officer declared that the EC\u2019s approval of the medicine was a \u201csignificant milestone\u201d for patients with disease.<\/p>\n At present, pegunigalsidase alfa is approved in the EU, Northern Ireland, and Great Britain for long-term ERT in adult patients who have been confirmed to have the disorder. The treatment is also approved in the US as a therapy for confirmed Fabry disease in adults.<\/p>\n Recommendation of Elfabrio\u00ae (pegunigalsidase alfa) means patients with Fabry disease in England will be able to access a new treatment option. The National Institute for Health and Care Excellence (NICE) has recommended Chiesi\u2019s Elfabrio\u00ae (pegunigalsidase alfa) for Fabry disease (alpha-galactosidase deficiency) in adults. A new Fabry disease treatment The agreement made in the NICE\u2019s Final […]<\/p>\n","protected":false,"gt_translate_keys":[{"key":"rendered","format":"html"}]},"author":2,"featured_media":20914,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[49],"tags":[],"acf":[],"gt_translate_keys":[{"key":"link","format":"url"}],"_links":{"self":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts\/20911"}],"collection":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/comments?post=20911"}],"version-history":[{"count":1,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts\/20911\/revisions"}],"predecessor-version":[{"id":20913,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts\/20911\/revisions\/20913"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/media\/20914"}],"wp:attachment":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/media?parent=20911"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/categories?post=20911"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/tags?post=20911"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}<\/div>\n
A new Fabry disease treatment<\/h2>\n
Positive regulatory activity for pegunigalsidase alfa in 2023<\/h2>\n
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