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01.02.2021 | AdisInsight Report

Lumasiran: First Approval

verfasst von: Lesley J. Scott, Susan J. Keam

Erschienen in: Drugs | Ausgabe 2/2021

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Abstract

Lumasiran (Oxlumo™) is a subcutaneously administered small interfering RNA (siRNA) targeting the mRNA for hydroxyacid oxidase 1 gene (HAO1; encodes glycolate oxidase) and was developed by Alnylam Pharmaceuticals for the treatment of primary hyperoxaluria type 1 (PH1). By silencing the gene encoding glycolate oxidase, lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of PH1. On 19 November 2020, lumasiran received its first global approval in the EU for the treatment of PH1 in all age groups. On 23 November 2020, lumasiran was approved in the USA for the treatment of adult and paediatric patients with PH1. This article summarizes the milestones in the development of lumasiran leading to this first approval.

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Titel: Lumasiran: First Approval
verfasst von: Lesley J. Scott
Susan J. Keam
Publikationsdatum: 01.02.2021
Verlag: Springer International Publishing
Erschienen in: Drugs / Ausgabe 2/2021
Print ISSN: 0012-6667
Elektronische ISSN: 1179-1950
DOI: https://doi.org/10.1007/s40265-020-01463-0

Springer Medizin

Abstract

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