New treatment for Fabry disease receives key recommendation

By Isabel Cameron

- Last updated on GMT

© Getty Images
© Getty Images

Related tags NICE Ert Rare disease Patient centricity

Chiesi, the biopharmaceutical and healthcare group, has announced that the National Institute for Health and Care Excellence (NICE) has recommended Elfabrio (pegunigalsidase alfa) as a treatment for Fabry disease in adults.

Pegunigalsidase alfa is a novel enzyme replacement therapy (ERT) administered via intravenous infusion every two weeks and delivers a modified version of the enzyme alpha-galactosidase A.

The therapy is designed for long-term treatment of adult patients with a confirmed diagnosis of Fabry disease, also known as alpha-galactosidase deficiency.

Fabry disease is a rare genetic disease affecting approximately 1,150 people in England. People with Fabry disease do not make enough of the enzyme, alpha-galactosidase A, which is needed to break down certain fatty acids.

When these fatty acids are not broken down properly, they can lead to a build-up, causing progressive damage to vital organs such as the heart, kidney and brain.

There is currently no cure for Fabry disease. However, available treatment options including ERT and chaperone therapy, can prevent progression of the disease and help to manage symptoms.

Dr Kamran Iqbal, head of medical affairs, global rare diseases, at Chiesi, said: “We are delighted that NICE has recommended pegunigalsidase alfa, bringing a new treatment option for people living with Fabry disease across England. Fabry 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.”

Bob Stevens, group chief executive of MPS Society, said: “On behalf of our Fabry community, the MPS Society welcomes the decision by NICE to make available the treatment pegunigalsidase alfa to our community, broadening the treatment options for those affected by Fabry. For people living with Fabry, it is vital that they are supported in living the lives they want and are able to make informed decisions about their treatment.”

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