Enzyme replacement therapies for Fabry disease

Published on 04 Jun 2024
Last Updated on 04 Jun 2024

Guidance Recommendations

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:

  1. Male patients with classical Fabry disease; or

  2. 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

Agalsidase 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.

Agalsidase alfa should be used in line with the additional clinical criteria listed in the Annex.

MAF assistance does not apply to agalsidase beta for treating Fabry disease.

Enzyme replacement therapies for Fabry disease (Published 4 Jun 24) [PDF, 137 KB]

PES Enzyme replacement therapies for treating Fabry disease (Published 4 Jun 24) [PDF, 112 KB]