{"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":"
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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

\n\"NICE\"NICE<\/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

A new Fabry disease treatment<\/h2>\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The agreement made in the NICE\u2019s Final Draft Guidance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

Positive regulatory activity for pegunigalsidase alfa in 2023<\/h2>\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