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.
Download detailed advice929KB (PDF)
Medicine details
- Medicine name:
- pegunigalsidase alfa (Elfabrio)
- SMC ID:
- SMC2591
- Indication:
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
- Full
- Status
- Not recommended
- Date advice published
- 13 November 2023