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Journal of Nephrology

Erschienen in:

01.02.2021 | Review

New drugs for the treatment of Anderson–Fabry disease

verfasst von: Sandro Feriozzi, Derralynn A. Hughes

Erschienen in: Journal of Nephrology | Ausgabe 1/2021

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Abstract

Enzyme replacement therapy (ERT) of the Anderson–Fabry disease (AFD) has changed the outcome of patients. However, ERT has some limitations: a restricted volume of distribution, requirement for intravenous access, and stimulation of the production of anti-drug antibodies. Studies of new drugs aiming to improve the clinical effectiveness and convenience of therapy have been reported. Migalastat, a pharmacological chaperone, increases available enzymate activity in patients with mutations amenable to the therapy, is now available for clinical practice. It is orally administered, and while clinical trial results are promising, long term real world follow up is awaited. PEGylated enzyme has a longer half-life and potentially reduced antigenicity, compared with standard preparations; investigation of whether a longer dosing interval is viable is under way. Moss-derived enzyme has a higher affinity for mannose receptors, and appears to have access to renal tissue. Substrate reduction therapy is based on reducing the catabolism processes of the glycosphingolipids, and is currently under investigation as monotherapy. Gene therapy has now been initiated in clinical trail of in vivo and ex vivo technologies with early results are emerging. ERT represents a certain milestone of therapy for AFD with Migalastat now a newly available option. Other agents in clinical trial prevent further potential opportunities to improve outcomes in AFD

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Titel: New drugs for the treatment of Anderson–Fabry disease
verfasst von: Sandro Feriozzi
Derralynn A. Hughes
Publikationsdatum: 01.02.2021
Verlag: Springer International Publishing
Erschienen in: Journal of Nephrology / Ausgabe 1/2021
Print ISSN: 1121-8428
Elektronische ISSN: 1724-6059
DOI: https://doi.org/10.1007/s40620-020-00721-4

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