The Scottish Medicines Consortium (SMC) has accepted pegungalsidase alfa (Elfabrio®) for the long-term treatment of adults with Fabry disease for restricted use. The restriction means that it is accepted for use to treat adults with Fabry disease who are experiencing symptoms and who would usually be offered an enzyme replacement therapy.
The Scottish Medicines Consortium (SMC) has now completed its assessment of pegungalsidase alfa (Elfabrio®) and, following review by the SMC executive, it advises NHS Boards and Area Drug and Therapeutics Committees (ADTCs) on its use in NHSScotland. Following a resubmission, the advice is summarised as follows:
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.
Bob Stevens, CEO of the MPS Society, says:
We are pleased that Scotland is now in line with the rest of the UK in regards to the treatment of Fabry.
If you have any questions regarding this treatment option, email Alison from the Support and Advocacy Team.
