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NICE recommends new Fabry disease treatment

by Samwel Doe Ouma
September 7, 2023
in News
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NICE recommends new Fabry disease treatment
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Protalix BioTherapeutics and Chiesi Farmaceutici have finally scored a marketing approval in Europe for PRX-102 (pegunigalsidase alfa), an enzyme replacement therapy for the rare, genetic, progressive disorder, which causes an accumulation of fatty deposits in the lysosomes and strikes roughly 1 in 50,000 people.

According to agreement seen by Health business magazine made in the NICE’s Final Draft Guidance -The Privately-held drugmaker -Chiesi has secured approval from the British medicines regulator for Elfabrio (pegunigalsidase alfa) in Fabry disease (FD).

The Italian firm has developed Elfabrio as a long-term enzyme replacement therapy for adults with FD, a serious lysosomal storage disorder which can result in impaired peripheral sensation and organ failure.

Chiesi is working with Israel’s Protalix BioTherapeutics (TASE: PLX) in the development of the therapy, the first and only enzyme replacement therapy of its type in this indication.

The authorization is based on results from a clinical program that has tested PRX-102 in more than 140 patients with up to 7.5 years of treatment.

A head-to-head trial pitting PRX-102 against Sanofi’s Fabrazyme (agalsidase beta) demonstrated noninferior efficacy in controlling the kidney disease that accompanies the disorder.

Fabrazyme, which was approved 20 years ago, is the longtime dominant drug in the Fabry disease market.

Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal α–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person’s body. Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the α–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3.

The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood and in the blood vessel walls. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure – particularly of the kidneys, but also of the heart and the cerebrovascular system.

Elfabrio is created with plant cells using recombinant DNA technology.

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Health Business

Health Business contains need-to-know features, news and case studies that explain the administrative and commercial issues affecting healthcare and hospital management. Health Business supports several high profile exhibitions - coverage of which is always timed for maximum impact. Regular topics include ICT, Finance/Funding, Facilities Management, Security, Health & Safety. Contributors range from government ministers through to top-level health administrators and association chairs.

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