Metazym

Metazym is a recombinant human arylsulfatase A (rhASA) enzyme replacement therapy developed for the treatment of metachromatic leukodystrophy (MLD). MLD is a rare lysosomal storage disorder characterized by a deficiency of the ARSA enzyme, which leads to the toxic accumulation of sulfatides in the central and peripheral nervous systems, causing progressive demyelination. Metazym is designed to replace the missing enzyme and facilitate the breakdown of accumulated sulfatides. Originally developed by Zymenex, the program was later acquired by Shire and subsequently Takeda. Clinical evaluation has included Phase 2 studies, such as those sponsored by Assistance Publique - Hôpitaux de Paris (AP-HP), which investigated the efficacy and safety of intravenous Metazym in patients with late infantile MLD who had previously undergone hematopoietic stem cell transplantation (HCT) to address residual neurological decline.

Brand names

Metazym

Other names

recombinant human arylsulfatase AMetazyme