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NICE approves its first ERT for treating Fabry disease

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We are pleased to announce that Pegunigalsidase alfa (Elfabrio®) has been recommended as an option by NICE (The National Institute for Health and Care Excellence) for treating adults with Fabry in England and Wales.

NICE committee members concluded that;

  • Pegunigalsidase alfa would be an additional ERT for people with and without an amenable mutation, and was a relevant comparator to exisiting treatments

  • Pegunigalsidase alfa was similarly clinically effective, as tolerable as the treatments used in the NHS, and costs less

Pegunigalsidase alfa (Elfabrio®), manufactured by pharmaceutical company Chiesi, is the first ERT treatment recommended for routine NHS use by NICE. This recommendation will give people with Fabry an additional treatment option.

Bob Stevens, Group Chief Executive of the MPS Society, said:

"On behalf of our Fabry community, we welcome 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.

The final draft guidance (FDG) document can be found on the NICE website here.

When NICE recommends a treatment, NHS England must make sure it is available within 3 months of the date of publication. For Wales, NHS Wales must usually provide funding and resources for it within 2 months of publication.


Notes for editors

Contact details

For further information or to arrange interviews, please contact the Communications team on 0345 389 9901 or email communications@mpssociety.org.uk.

About MPS Society

Founded in 1982, by Christine Lavery, MBE, the Society for Mucopolysaccharide Diseases, or MPS Society, is the only registered charity providing professional support to individuals and families affected by the rare, life-limiting genetic diseases: MPS, Fabry and related disease in the UK. The MPS Society is transforming the lives of those affected and supports over 1,500 people per year.

About Chiesi Group

Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.

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