The NICE committee concluded that the treatment would be an additional ERT for patients with and without an amenable mutation and was a relevant comparator to existing treatments.
Elfabrio is a novel enzyme replacement therapy (ERT) that is administered via intravenous infusion every two weeks. The treatment works to deliver a modified version of the enzyme a-galactosidase for those with a confirmed diagnosis of Fabry disease.
The recommendation was based on results from phase 3 clinical trials, which showed that Elfabrio was generally well tolerated and was similarly clinically effective and as tolerable as the current treatments, agalsidase alfa and migalastat, used in the NHS…
