NICE recommends Elfabrio®▼(pegunigalsidase alfa) for adults with Fabry Disease in Final Draft Guidance

• Pegunigalsidase alfa is a novel enzyme replacement therapy (ERT).
• Fabry disease, a rare genetic condition, affects around 1,150 people in England.

MANCHESTER, UK, 1st September 2023 – Chiesi, the international research-focused biopharmaceutical and healthcare group, today announced that 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known as alpha-galactosidase deficiency) in adults under the terms of a confidential commercial agreement in Final Draft Guidance.[1]

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.[2],3 It is indicated for long-term treatment of adult patients with a confirmed diagnosis of Fabry disease.2

“We are delighted that NICE has recommended pegunigalsidase alfa, bringing a new treatment option for people living with Fabry disease across England,” said Dr Kamran Iqbal, Head of Medical Affairs, Global Rare Diseases, Chiesi UK&I. "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.”

Fabry disease is a rare genetic disease affecting approximately 1,150 people in England.[3] People with Fabry disease do not make enough of the enzyme, alpha-galactosidase A, which is needed to break down certain fatty acids.[4] 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.4 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.1

“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,” said Bob Stevens, Group Chief Executive, MPS Society. “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.”

[1] National Institute for Health and Care Excellence. Pegunigalsidase alfa for treating Fabry disease. Final Draft Guidance. Available at https://www.nice.org.uk/guidance/gid-ta10790/documents/674. Last accessed September 2023.

[2] Electronic Medicines Compendium. Pegunigalsidase alfa Summary of Product Characteristics. Available at https://www.medicines.org.uk/emc/product/14960/smpc#gref. Last accessed September 2023.

[3] National Institute for Health and Care Excellence. Pegunigalsidase alfa for treating Fabry disease. Final Scope. Available at https://www.nice.org.uk/guidance/gid-ta10790/documents/final-scope. Last accessed September 2023.

[4] Bernat et al. eP149: Safety and efficacy of pegunigalsidase alfa, every 4 weeks, in Fabry disease: Results from the phase 3, open-label, BRIGHT study (Abstract). Genetics in Medicine. 2022; 27(3): S91-92.