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Condition update published on

SMC do not recommend pegunigalsidase alfa (Elfabrio®) for adult Fabry patients


SMC do not recommend pegunigalsidase alfa (Elfabrio®) for adult Fabry patients.

The Scottish Medicines Consortium (SMC), the Scottish drug decision making body, have publicly released their decision today not to recommend pegunigalsidase alfa (Elfabrio®) for the long-term treatment of adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).

The SMC acknowledged that pegunigalsidase alfa (Elfabrio®) had a similar impact on kidney function when compared to other Enzyme Replacement Therapies for Fabry disease. However, they did not feel that additional clinical and economic evidence was sufficiently robust.

Pegunigalsidase alfa (Elfabrio®) is manufactured by pharmaceutical company Chiesi who commented:

"We remain committed to ensuring access for all eligible people living with Fabry disease who may benefit from this treatment, and we will continue to work closely with the SMC to resubmit at the earliest opportunity."

Approval by the SMC would have brought treatment choices for Scottish Fabry patients in line with other parts of the UK. The MPS Society will continue to work closely with all parties to ensure that our members in Scotland have equitable access to therapies.

A detailed summary of the advice is available from the SMC website.

Notes for editors

Contact details

For further information or to arrange interviews, please contact the Communications team on 0345 389 9901 or email

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 conditions in the UK. The MPS Society is transforming the lives of those affected and supports over 1,750 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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