following a full submission assessed under the orphan equivalent medicine process:
pegunigalsidase alfa (Elfabrio®) is not recommended for use within NHSScotland.
Indication Under Review: for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry Disease (deficiency of alpha-galactosidase).
In a two-year, double-blind, randomised, phase III study, pegunigalsidase alfa appeared to have a similar annualised change in estimated glomerular filtration rate (eGFR) compared with an alternative enzyme replacement therapy.
The company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
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- Medicine name:
- pegunigalsidase alfa (Elfabrio)
- SMC ID:
Long-term enzyme replacement therapy (ERT) in adult patients with confirmed diagnosis of Fabry disease (FD) (deficiency of alpha galactosidase).
- Pharmaceutical company
- Chiesi Limited
- BNF chapter
- Nutrition and blood
- Submission type
- Not recommended
- Date advice published
- 13 November 2023