following a resubmission:
pegunigalsidase alfa (Elfabrio®) is accepted for restricted 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).
SMC restriction: for use in adults with symptomatic Fabry disease who would usually be offered an enzyme replacement therapy.
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.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
Medicine details
- Medicine name:
- pegunigalsidase alfa (Elfabrio)
- SMC ID:
- SMC2665
- 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
- Resubmission
- Status
- Restricted
- Date advice published
- 08 July 2024