UK's NICE backs Elfabrio for Fabry disease

1 September 2023

Following swiftly on regulatory approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), privately-held Italian drugmaker Chiesi has now also won backing from the health technology assessor for its Fabry disease drug.

The National Institute for Health and Care Excellence (NICE) has recommended Elfabrio (pegunigalsidase alfa) as an option for treating Fabry disease (also known as alpha-galactosidase deficiency) in adults under the terms of a confidential commercial agreement in Final Draft Guidance.

Pegunigalsidase alfa is a novel enzyme replacement therapy (ERT) administered via intravenous infusion every two weeks and delivers a modified version of the enzyme α-galactosidase A. It is indicated for long-term treatment of adult patients with a confirmed diagnosis of Fabry disease.

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