04/06/2024 Enzyme replacement therapies for Fabry disease
The Ministry of Health’s Drug Advisory Committee has recommended:
Agalsidase alfa 3.5 mg/3.5 mL concentrate for solution for infusion as an enzyme replacement therapy in patients with Fabry disease. The treatment may be initiated only in:
a) Male patients with classical Fabry disease; or
b) Male patients with non-classical Fabry disease, or female patients with classical or non-classical Fabry disease, who have signs/symptoms of organ involvement (e.g. kidney, heart or nervous system) consistent with Fabry disease and which are not fully explained by other pathology.
Funding status
RAgalsidase alfa 3.5 mg/3.5 mL concentrate for solution for infusion is recommended for inclusion on the Medication Assistance Fund (MAF) for the abovementioned indication from 1 August 2024.
RAgalsidase alfa should be used in line with the additional clinical criteria listed in the Annex.
NRMAF assistance does not apply to agalsidase beta for treating Fabry disease.